Entries |
Document | Title | Date |
20080221317 | siRNA targeting cystic fibrosis transmembrane conductance regulator (CFTR) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to CFTR. | 09-11-2008 |
20080242851 | MODIFIED POLYNUCLEOTIDES FOR REDUCING OFF-TARGET EFFECTS IN RNA INTERFERENCE - Methods and compositions for performing RNA interference with decreased off-target effects are provided. The methods and compositions permit effective and efficient applications of RNA interference to applications such as diagnostics and therapeutics through the use of modifications to the siRNA. Uniquely modified siRNAs have been developed that reduce off-target effects incurred in gene-silencing. The modifications comprise 2′-O-alkyl or mismatch modification(s) at specific positions on the sense and/or antisense strands. | 10-02-2008 |
20080249294 | RNA Interference Mediated Inhibition of Gene Expression Using Short Interfering Nucleic Acid (siNA) - This invention relates to compounds, compositions, and methods useful for modulating gene expression using short interfering nucleic acid (siNA) molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of genes, such as expressed pseudogenes associated with the maintenance or development of diseases, disorders, traits, and conditions in a subject or organism. The invention also provides small nucleic acid molecules with reduced or attenuated immunostimulatory properties and methods for designing and synthesizing such small nucleic acid molecules having improved toxicologic properties while retaining RNAi activity. | 10-09-2008 |
20080269474 | Novel shRNA molecules and methods of use thereof - The present invention relates to certain novel shRNA molecules and methods of use thereof. According to certain embodiments of the present invention, methods for reducing the expression level of a target gene are provided. Such methods generally comprise providing a cell with one or more precursor nucleic acid sequences that encode two or more RNA molecules. A first RNA molecule comprises a double stranded sequence, which includes a guide strand sequence that is complementary to a portion of an mRNA transcript encoded by the target gene. In addition, a second RNA molecule comprises a second double stranded sequence, which includes a second guide strand sequence that is partially complementary to a portion of the mRNA transcript encoded by the target gene. Preferably, the second guide strand sequence comprises one or more bases that are mismatched with a nucleic acid sequence of the mRNA transcript encoded by the target gene. | 10-30-2008 |
20080300395 | siRNA targeting vascular endothelial growth factor receptor 1 (VEGFR1) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to VEGFR1. | 12-04-2008 |
20080319180 | siRNA targeting protein kinase N-3 (PKN-3) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to PKN-3. | 12-25-2008 |
20080319181 | Method of Making an Artificial Nuclease for Anti-viral, Anti-bacterial Applications - A method of making a macrocyclic chelator comprising converting Co(II)Cl | 12-25-2008 |
20090005547 | siRNa targeting neuropilin 1 (NRP1) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to NRP1. | 01-01-2009 |
20090005548 | siRNA targeting nuclear receptor interacting protein 1 (NRIP1) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to NRIP1. | 01-01-2009 |
20090018321 | METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION BY DOUBLE-STRANDED RNA - The invention provides compositions and methods for selectively reducing the expression of a gene product from a desired target gene, as well as treating diseases caused by expression of the gene. The method involves introducing into the environment of a cell an amount of a double-stranded RNA (dsRNA) such that a sufficient portion of the dsRNA can enter the cytoplasm of the cell to cause a reduction in the expression of the target gene. The dsRNA has a first oligonucleotide sequence that is between 26 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of from about 19 to about 23 nucleotides is complementary to a nucleotide sequence of the RNA produced from the target gene. | 01-15-2009 |
20090023907 | siRNA targeting kinesin spindle protein (KSP) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to KSP. | 01-22-2009 |
20090023908 | siRNA targeting ribosomal protein S2 (RPS2) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to RPS2. | 01-22-2009 |
20090030190 | siRNA targeting 1-acylglycerol-3-phosphate O-acyltransferase 2 (AGPAT2) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to AGPAT2. | 01-29-2009 |
20090036661 | METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION BY DOUBLE-STRANDED RNA - The invention is directed to compositions and methods for selectively reducing the expression of a gene product from a desired target gene in a cell, as well as for treating diseases caused by the expression of the gene. More particularly, the invention is directed to compositions that contain double stranded RNA (“dsRNA”), and methods for preparing them, that are capable of reducing the expression of target genes in eukaryotic cells. The dsRNA has a first oligonucleotide sequence that is between 25 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of at least 19 nucleotides is sufficiently complementary to a nucleotide sequence of the RNA produced from the target gene to trigger the destruction of the target RNA by the RNAi machinery. | 02-05-2009 |
20090036662 | Hypoxia-regulated genes - According to the present invention, purified, isolated and cloned nucleic acid polynucleotide encoding hypoxia-regulating genes and the proteins thereof and antibodies directed against the proteins which have sequences as set forth in SEQ ID No:1, SEQ ID No:2, SEQ ID No:3, SEQ ID No:4, SEQ ID No:5 and SEQ ID No:6 are provided. The present invention further provides transgenic animals and cell lines as well as knock-out organisms of these sequences. The present invention further provides methods of regulating angiogenesis or apoptosis or regulating response to hypoxic conditions in a patient in need of such treatment. The present invention also provides a method of diagnosing the presence of ischemia in a patient including the steps of analyzing a bodily fluid or tissue sample from the patient for the presence or gene product of at least one expressed gene (up-regulated) as set forth in the group comprising SEQ ID No:2; SEQ ID No:3; SEQ ID No:4; SEQ ID No:5; and SEQ ID No:6 and where ischemia is determined if the up-regulated gene or gene product is ascertained. | 02-05-2009 |
20090043084 | siRNA targeting minichromosome maintenance deficient 3 (MCM3) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to MCM3. | 02-12-2009 |
20090043085 | METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION BY DOUBLE-STRANDED RNA - The invention is directed to compositions and methods for selectively reducing the expression of a gene product from a desired target gene in a cell, as well as for treating diseases caused by the expression of the gene. More particularly, the invention is directed to compositions that contain double stranded RNA (“dsRNA”), and methods for preparing them, that are capable of reducing the expression of target genes in eukaryotic cells. The dsRNA has a first oligonucleotide sequence that is between 25 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of at least 19 nucleotides is sufficiently complementary to a nucleotide sequence of the RNA produced from the target gene to trigger the destruction of the target RNA by the RNAi machinery. | 02-12-2009 |
20090048435 | Oligonucleotide modulation of cell adhesion - Compositions comprising oligonucleotides which are specifically hybridizable with nucleic acids encoding cellular adhesion molecules intracellular adhesion molecule-1 (ICAM-1), vascular cell adhesion molecule-1 (VCAM-1) and endothelial leukocyte adhesion molecule-1 (ELAM-1) are disclosed. A series of double stranded RNA molecules targeting human ICAM-1 were designed and inhibition of RNA was measured. Oligonucleotides targeted to ICAM were effective in reducing airway hyperresponsiveness in mouse and monkey asthma models. | 02-19-2009 |
20090082556 | siRNA targeting TATA box binding protein (TBP)-associated factor (TAF1) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for TAF1. | 03-26-2009 |
20090088562 | Oligonucleotide analogs having cationic intersubunit linkages - Morpholino oligomers containing both uncharged and cationic intersubunit linkages are provided. The oligomers are oligonucleotide analogs containing predetermined sequences of base-pairing moieties. The presence of the cationic intersubunit linkages in the oligomers, typically at a level of about 10-50% of total linkages, provides enhanced antisense activity, in various antisense applications, relative to the corresponding uncharged oligomers. Also provided are such oligomers conjugated to peptide transporter moieties, where the transporters are preferably composed of arginine subunits, or arginine dimers, alternating with neutral amino acid subunits. | 04-02-2009 |
20090088563 | siRNA targeting Transducin (beta)-like 3 (TBL3) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for TBL3. | 04-02-2009 |
20090105467 | Antisense oligonucleotide constructs based on beta-arabinofuranose and its analogues - The present invention relates to modified oligonucleotide therapeutic agents to selectively prevent gene transcription and expression in a sequence-specific manner. In particular, this invention relates to the selective inhibition of protein biosynthesis via antisense strategy using oligonucleotides constructed from arabinonucleotide or modified arabinonucleotide residues. More particularly this invention relates to the use of antisense oligonucleotides having arabinose sugars to hybridize to complementary RNA such as cellular messenger RNA, viral RNA, etc. | 04-23-2009 |
20090111977 | ANTISENSE COMPOUND FOR INDUCING IMMUNOLOGICAL TOLERANCE - A method and conjugate for selectively killing antigen-activated T cells are disclosed. The conjugate is composed of a substantially uncharged antisense compound targeted against the human cFLIP protein, and a reverse TAT (rTAT) polypeptide coupled covalently to the antisense compound. The rTAT polypeptide is effective to produce selective uptake of the conjugate into antigen-activated T cells, relative to the uptake of the conjugate into non-activated T cells. The cFLIP antisense compound causes activation induced cell death (AICD) of activated lymphocytes. The method is useful in treating transplantation rejection and autoimmune conditions. | 04-30-2009 |
20090118489 | siRNA targeting nucleoporin 62kDa (Nup62) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for Nup62. | 05-07-2009 |
20090149643 | Aptamers to von Willebrand factor and their use as thrombotic disease therapeutics - The invention relates generally to the field of nucleic acids and more particularly to aptamers capable of binding to von Willebrand Factor useful as therapeutics in and diagnostics of thrombotic diseases and/or other diseases or disorders in which von Willebrand Factor mediated platelet aggregation has been implicated. The invention further relates to materials and methods for the administration of aptamers capable of binding to von Willebrand Factor. | 06-11-2009 |
20090149644 | siRNA Targeting KRAS - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs directed to silencing KRAS, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. | 06-11-2009 |
20090156797 | siRNA Targeting Hypoxia-inducible Factor 1 - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed. | 06-18-2009 |
20090163701 | siRNA targeting tumor necrosis factor receptor superfamily member 1A - Efficient sequence specific gene silencing of siRNA targeting tumor necrosis factor receptor superfamily member 1A is possible through the use of siRNA technology. By selecting particular siRNAs by rationale design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. | 06-25-2009 |
20090163702 | siRNA targeting Myeloid cell leukemia sequence 1 - Efficient sequence specific gene silencing of myeloid cell leukemia sequence 1 is possible through the use of siRNA technology. By selecting particular siRNAs directed to myeloid cell leukemia sequence 1 by rationale design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. | 06-25-2009 |
20090176977 | LNA MODIFIED PHOSPHOROTHIOLATED OLIGONUCLEOTIDES - The current invention provides oligonucleotides which comprise a dinucleotide consisting of a 5′ locked nucleic acid (LNA), a phosphorothioate internucleoside linkage bond to a 3′ RNA or RNA analogue. The dinucleotide reduces the strength of hybridization of the oligonucleotide to a complementary nucleic acid target. The modification can be used to modulate hybridisation properties in both single stranded oligonucleotides and in double stranded siRNA complexes, particularly in oligonucleotides where the use of LNA results in excessively strong hybridisation properties. | 07-09-2009 |
20090203892 | Retrotransposon Inhibition in Therapy - RNA interference is useful in the treatment of cancerous lesions, wherein the RNA recognises a portion of at least one LINE-I repeat element. | 08-13-2009 |
20090203893 | ANTISENSE COMPOUNDS HAVING ENHANCED ANTI-MICRORNA ACTIVITY - Antisense compounds, compositions and methods are provided for modulating the levels expression, processing and function of miRNAs. The antisense compounds exhibit enhanced anti-miRNA activity. Further provided are methods for enhancing the inhibitory activity of an antisense compound targeting a miRNA, comprising incorporating stability enhancing sugar modifications into the antisense compounds. | 08-13-2009 |
20090203894 | Composition and methods of RNAi therapeutics for treatment of cancer and other neovascularization diseases - Compositions and methods are provided for treatment of diseases involving unwanted neovascularization (NV). The invention provides treatments that control NV through selective inhibition of pro-angiogenic biochemical pathways, including inhibition of the VEGF pathway gene expression and inhibition localized at pathological NV tissues. Tissue targeted nanoparticle compositions comprising polymer conjugates and nucleic acid molecules that induce RNA interference (RNAi) are provided. The nanoparticle compositions of the invention can be used alone or in combination with other therapeutic agents such as VEGF pathway antagonists. The compositions and methods can be used for the treatment of NV diseases such as cancer, ocular disease, arthritis, and inflammatory diseases. | 08-13-2009 |
20090203895 | siRNA targeting cyclin-dependent kinase 4 (CDK4) - Efficient sequence specific gene silencing for cyclin-dependent kinase 4 is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed. | 08-13-2009 |
20090203896 | MODULATION OF SURVIVIN EXPRESSION - Compounds and compositions are provided for modulating the expression of survivin. The compounds, exemplified by those acting through an RNAi antisense mechanism of action, include double-stranded and single-stranded constructs, as well as siRNAs, canonical siRNAs, blunt-ended siRNAs and single-stranded antisense RNA compounds. Methods of using these compounds for modulation of survivin expression and for treatment of diseases associated with expression of survivin are provided. | 08-13-2009 |
20090240043 | RNAi MODULATION OF RSV AND THERAPEUTIC USES THEREOF - The present invention is based on the in vivo demonstration that RSV can be inhibited through intranasal administration of iRNA agents as well as by parenteral administration of such agents. Further, it is shown that effective viral reduction can be achieved with more than one virus being treated concurrently. Based on these findings, the present invention provides general and specific compositions and methods that are useful in reducing RSV mRNA levels, RSV protein levels and viral titers in a subject, e.g., a mammal, such as a human. These findings can be applied to other respiratory viruses. | 09-24-2009 |
20090264636 | CONJUGATES AND COMPOSITIONS FOR CELLULAR DELIVERY - This invention features conjugates, degradable linkers, compositions, methods of synthesis, and applications thereof, including cholesterol, folate, galactose, galactosamine, N-acetyl galactosamine, PEG, phospholipid, peptide and human serum albumin (HSA) derived conjugates of biologically active compounds, including antibodies, antivirals, chemotherapeutics, peptides, proteins, hormones, nucleosides, nucleotides, non-nucleosides, and nucleic acids including enzymatic nucleic acids, DNAzymes, allozymes, antisense, dsRNA, siNA, siRNA, triplex oligonucleotides, 2,5-A chimeras, decoys and aptamers. | 10-22-2009 |
20090281297 | siRNA Targeting v-myc myelocytomatosis viral oncogene homolog (MYC) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to MYC. | 11-12-2009 |
20090281298 | OLIGONUCLEOTIDES COMPRISING A MODIFIED OR NON-NATURAL NUCLEOBASE - One aspect of the present invention relates to a double-stranded oligonucleotide comprising at least one non-natural nucleobase. In certain embodiments, the non-natural nucleobase is difluorotolyl, nitroindolyl, nitropyrrolyl, or nitroimidazolyl. In a preferred embodiment, the non-natural nucleobase is difluorotolyl. In certain embodiments, only one of the two oligonucleotide strands comprising the double-stranded oligonucleotide contains a non-natural nucleobase. In certain embodiments, both of the oligonucleotide strands comprising the double-stranded oligonucleotide independently contain a non-natural nucleobase. In certain embodiments, the oligonucleotide strands comprise at least one modified sugar moiety. Another aspect of the present invention relates to a single-stranded oligonucleotide comprising at least one non-natural nucleobase. In a preferred embodiment, the non-natural nucleobase is difluorotolyl. In certain embodiments, the ribose sugar moiety that occurs naturally in nucleosides is replaced with a hexose sugar, polycyclic heteroalkyl ring, or cyclohexenyl group. In certain embodiments, at least one phosphate linkage in the oligonucleotide has been replaced with a phosphorothioate linkage. | 11-12-2009 |
20090299045 | RNA Interference Mediated Inhibition Of Interleukin and Interleukin Gene Expression Using Short Interfering Nucleic Acid (siNA) - This invention relates to compounds, compositions, and methods useful for modulating interleukin and/or interleukin receptor gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of interleukin and/or interleukin receptor gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of interleukin and/or interleukin receptor genes, such as IL-1, IL-2, IL-3, IL-4, IL-5, IL-6, IL-7, IL-8, IL-9, IL-10, IL-11, IL-12, IL-13, IL-14, IL-15, IL-16, IL-17, IL-18, IL-19, IL-20, IL-21, IL-22, IL-23, IL-24, IL-25, IL-26, and IL-27 genes and IL-1R, IL-2R, IL-3R, IL-4R, IL-5R, IL-6R, IL-7R, IL8R, IL-9R, IL-10R, IL-11R, IL-12R, IL-13R, IL-14R, IL-15R, IL-16R, IL-17R, IL-18R, IL19R, IL-20R, IL-21R, IL-22R, IL-23R, IL-24R, IL-25R, IL-26R, and IL-27R. Such small nucleic acid molecules are useful, for example, for treating, preventing, inhibiting, or reducing cancer, inflammatory, respiratory, autoimmune, cardiovascular, neurological, and/or proliferative diseases, disorders, or conditions in a subject or organism, and for any other disease, trait, or condition that is related to or will respond to the levels of interleukin and/or interleukin receptor in a cell or tissue, alone or in combination with other treatments or therapies. | 12-03-2009 |
20090306356 | siRNA Targeting TNFalpha - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to TNFα. | 12-10-2009 |
20090306357 | COMPOUNDS AND METHODS FOR MODULATING EXPRESSION OF GCCR - The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing. | 12-10-2009 |
20100004434 | METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION BY DOUBLE-STRANDED RNA - The invention is directed to compositions and methods for selectively reducing the expression of a gene product from a desired target gene in a cell, as well as for treating diseases caused by the expression of the gene. More particularly, the invention is directed to compositions that contain double stranded RNA (“dsRNA”), and methods for preparing them, that are capable of reducing the expression of target genes in eukaryotic cells. The dsRNA has a first oligonucleotide sequence that is between 25 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of at least 19 nucleotides is sufficiently complementary to a nucleotide sequence of the RNA produced from the target gene to trigger the destruction of the target RNA by the RNAi machinery. | 01-07-2010 |
20100004435 | METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION BY DOUBLE-STRANDED RNA - The invention provides compositions and methods for selectively reducing the expression of a gene product from a desired target gene, as well as treating diseases caused by expression of the gene. The method involves introducing into the environment of a cell an amount of a double-stranded RNA (dsRNA) such that a sufficient portion of the dsRNA can enter the cytoplasm of the cell to cause a reduction in the expression of the target gene. The dsRNA has a first oligonucleotide sequence that is between 26 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of from about 19 to about 23 nucleotides is complementary to a nucleotide sequence of the RNA produced from the target gene. | 01-07-2010 |
20100010206 | siRNA Targeting Hypoxia-Inducible Factor 1 - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed. | 01-14-2010 |
20100010207 | RNA INTERFERENCE MEDIATING SMALL RNA MOLECULES - Double-stranded RNA (dsRNA) induces sequence-specific post-transcriptional gene silencing in many organisms by a process known as RNA interference (RNAi). Using a | 01-14-2010 |
20100016573 | siRNA targeting cyclin-dependent kinase inhibitor 1B (p27, Kip1) (CDKN1B) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for CDKN1B. | 01-21-2010 |
20100022763 | siRNA targeting kinase insert domain receptor (KDR) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for KDR. | 01-28-2010 |
20100036106 | High-Affinity RNA Aptamer Molecule Against Glutathione-S-Transferase Protein - The present invention provides a “nucleic acid adaptor molecule” having specific binding affinity to a GST protein portion serving as an N-terminal fusion partner in a fusion protein consisting of the GST protein and a protein of interest. A “nucleic acid adaptor molecule against a GST protein” according to the present invention is an RNA aptamer molecule having any of the following nucleotide sequences I to III: | 02-11-2010 |
20100036107 | RNA INTERFERENCE COMPOSITIONS AND METHODS - The invention provides isolated nucleic acids. For example, the invention provides isolated nucleic acids having at least one strand with both sense and antisense sequences that are complementary to each other. The invention also provides isolated nucleic acids having at least one strand that is a template for both sense and antisense sequences that are complementary to each other. In addition, the invention provides cells, viruses, and transgenic animals (e.g., transgenic non-human animals) containing one or more of the isolated nucleic acids provided herein as well as methods for using one or more of the isolated nucleic acids provided herein to reduce the level of an RNA (e.g., an mRNA) within a cell. | 02-11-2010 |
20100069622 | siRNA targeting amyloid beta (A4) precursor protein (APP) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for APP. | 03-18-2010 |
20100093987 | Inhibitor Nucleic Acids - The present invention provides methods and compositions for attenuating expression of a target gene in vivo. In general, the method includes administering RNAi constructs (such as small-interfering RNAs (i.e., siRNAs) that are targeted to particular mRNA sequences, or nucleic acid material that can produce siRNAs in a cell), in an amount sufficient to attenuate expression of a target gene by an RNA interference mechanism. In particular, the RNAi constructs may include one or more modifications to improve serum stability, cellular uptake and/or to avoid non-specific effect. In certain embodiments, the RNAi constructs contain an aptamer portion. The aptamer may bind to human serum albumin to improve serum half life. The aptamer may also bind to a cell surface protein that improves uptake of the construct. | 04-15-2010 |
20100113760 | siRNA targeting myeloid differentiation primary response gene (88) (MYD88) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for MYD88. | 05-06-2010 |
20100113761 | siRNA targeting beta secretase (BACE) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for BACE. | 05-06-2010 |
20100121043 | ENVIRONMENT-RESPONDING siRNA CARRIER USING DISULFIDE-CROSS-LINKED POLYMERIC MICELLE - Disclosed is a siRNA-encapsulating polymeric micelle complex (a polyion complex) having high monodispersibility and structural stability and excellent in the ability of transporting siRNA into a cell. Further disclosed are a nucleic acid delivery device, a nucleic acid delivery kit, a pharmaceutical composition, and a gene therapy agent, each of which uses the complex. The polyion complex is characterized by comprising: a block copolymer composed of a polyethylene glycol moiety and a polycation moiety having a thiol group as a side chain at the terminus; and siRNA. | 05-13-2010 |
20100145038 | RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating gene expression using short interfering nucleic acid (siNA) molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of genes, such as expressed pseudogenes associated with the maintenance or development of diseases, disorders, traits, and conditions in a subject or organism. | 06-10-2010 |
20100145039 | siRNA targeting nucleoporin 62kDa (Nup62) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for Nup62 | 06-10-2010 |
20100179309 | MODIFIED iRNA AGENTS - The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose. The inclusion of such a monomer can allow for modulation of a property of the iRNA agent into which it is incorporated, e.g., by using the non-ribose moiety as a point to which a ligand or other entity, e.g., a carbohydrate; or a steroid, e.g., cholesterol, which is optionally substituted with at least one carbohydrate. is directly, or indirectly, tethered. The invention also relates to methods of making and using such modified iRNA agents. | 07-15-2010 |
20100190971 | siRNA Targeting Diacylglycerol O-Acyltransferase Homolog 2 (DGAT2) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for DGAT2. | 07-29-2010 |
20100197901 | DEPROTECTION AND PURIFICATION OF OLIGONUCLEOTIDES AND THEIR DERIVATIVES - Method for synthesis, deprotection, and/or purification of nucleic acid molecules, such as oligonucleotides comprising one or more ribonucleotides. Such nucleic acid molecules include siRNA, dsRNA, ribozymes, antisense, and aptamers. | 08-05-2010 |
20100216981 | ANTIVIRAL AGENT - An object of the present invention is to provide a viral disease control agent which has a mechanism of action different from conventional one as a substitute for existing viral disease control methods and is used in more practical and safer manners. The present invention utilizes a compound having inhibitory activity on the binding of a substance α to a PTGS suppressor protein, wherein the substance α has a property of inducing PTGS and a property of binding to the PTGS suppressor protein and shows a decrease in the property of inducing PTGS upon binding to the PTGS suppressor protein. | 08-26-2010 |
20100216982 | POLYCYCLIC SUGAR SURROGATE-CONTAINING OLIGOMERIC COMPOUNDS AND COMPOSITIONS FOR USE IN GENE MODULATION - Compositions comprising first and second oligomers are provided wherein at least a portion of the first oligomer is capable of hybridizing with at least a portion of the second oligomer, at least a portion of the first oligomer is complementary to and capable of hybridizing to a selected target nucleic acid, and at least one of the first or second oligomers includes a modification comprising a polycyclic sugar surrogate. Oligomer/protein compositions are also provided comprising an oligomer complementary to and capable of hybridizing to a selected target nucleic acid and at least one protein comprising at least a portion of an RNA-induced silencing complex (RISC), wherein at least one nucleoside of the oligomer has a polycyclic sugar surrogate modification. | 08-26-2010 |
20100228018 | RNA INTERFERENCE MEDIATED INHIBITION OF PROLIFERATING CELL NUCLEAR ANTIGEN (PCNA) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating PCNA gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of PCNA gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of PCNA genes. The small nucleic acid molecules are useful in the treatment of cancer or restenosis or other proliferative diseases, disorders, or conditions. | 09-09-2010 |
20100234582 | siRNA targeting gremlin - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to CKSF1B1. | 09-16-2010 |
20100234583 | siRNA target hypoxia-inducible factor 1 - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed. | 09-16-2010 |
20100240881 | THERAPEUTIC COMPOSITIONS - This application relates to therapeutic siRNA agents and methods of making and using the agents. | 09-23-2010 |
20100267941 | IRNA AGENTS WITH BIOCLEAVABLE TETHERS - The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose that further includes a tether having one or more linking groups, in which at least one of the linking groups is a cleavable linking group. The tether in turn can be connected to a selected moiety, e.g., a ligand, e.g., a targeting or delivery moiety, or a moiety which alters a physical property. The cleavable linking group is one which is sufficiently stable outside the cell such that it allows targeting of a therapeutically beneficial amount of an iRNA agent (e.g., a single stranded or double stranded iRNA agent), coupled by way of the cleavable linking group to a targeting agent—to targets cells, but which upon entry into a target cell is cleaved to release the iRNA agent from the targeting agent. | 10-21-2010 |
20100273997 | Ribozyme to cleave coronavirus gene - Provided is a ribozyme to cleave a coronavirus gene and a therapeutic agent for a coronavirus infectious disease. A common base sequence in coronaviruses such as SARS-CoV and MHV was searched to design a ribozyme including a base sequence complementary thereto. Moreover, a therapeutic agent for a coronavirus infectious disease including such ribozyme was obtained. | 10-28-2010 |
20100273998 | METHODS AND COMPOSITIONS FOR MODULATING TISSUE FACTOR - The present invention features nucleic acid molecules, specifically short interfering RNA molecules, that are able to modulate the expression of TF and methods for using such molecules for the treatment of coagulative disorders and for the prevention and treatment of cancer. | 10-28-2010 |
20100286384 | REPLIKIN PEPTIDES IN RAPID REPLICATION OF GLIOMA CELLS AND IN INFLUENZA EPIDEMICS - Peptides of influenza virus hemagglutinin protein and | 11-11-2010 |
20100286385 | Anti-MicroRNA Oligonucleotide Molecules - The invention relates to isolated anti-microRNA molecules. In another embodiment, the invention relates to an isolated microRNA molecule. In yet another embodiment, the invention provides a method for inhibiting microRNP activity in a cell. | 11-11-2010 |
20100292454 | NEURONAL REGENERATION PROMOTING AGENT - It is an object of the present invention to provide a novel neuronal regeneration promoting agent, particularly a neuronal regeneration promoting agent having an inhibitory effect on glial scar formation. The present invention provides a neuronal regeneration promoting agent which comprises an inhibitor of a bone morphogenetic protein type 1A receptor (BMPR1A) as an active ingredient. | 11-18-2010 |
20100292455 | MODIFIED iRNA AGENTS - The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose. The inclusion of such a monomer can allow for modulation of a property of the iRNA agent into which it is incorporated, e.g., by using the non-ribose moiety as a point to which a ligand or other entity, e.g., a lipophilic moiety. e.g., cholesterol, is is directly, or indirectly, tethered. The invention also relates to methods of making and using such modified iRNA agents. | 11-18-2010 |
20100292456 | RNA INTERFERENCE MEDIATING SMALL RNA MOLECULES - Double-stranded RNA (dsRNA) induces sequence-specific post-transcriptional gene silencing in many organisms by a process known as RNA interference (RNAi). Using a | 11-18-2010 |
20100317840 | hnRNP K EXPRESSION-INHIBITING COMPOUND AND SIRNA SEQUENCE THEREOF - The present invention discloses an hnRNP K expression-inhibiting compound and a siRNA sequence thereof, wherein a siRNA sequence partially or completely complementary to the sequence of hnRNP K is used to inhibit hnRNP K expression, whereby is effectively reduced the survival rate of cancer cells in an anoxic environment. | 12-16-2010 |
20100331538 | CARBOCYCLIC ALPHA-L-BICYCLIC NUCLEIC ACID ANALOGS - The present invention provides novel carbocyclic α-L-bicyclic nucleosides and oligomeric compounds comprising at least one of these carbocyclic α-L-bicyclic nucleosides. The carbocyclic α-L-bicyclic nucleosides are useful for enhancing one or more properties of the oligomeric compounds they are incorporated into including nuclease resistance. | 12-30-2010 |
20110009606 | Synthesis of 2',3' - and 3',5' -cyclic phosphate mono-and oligonucleotides - The invention provides a novel method of 2′,3′-cyclic phosphate and phosphorothioate of mono and oligonucleotide synthesis. The invention also provides a novel method of the synthesis of 3′,5′-cyclic phosphate and phosphorothioate mononucleotide. The invention also envisions providing kits comprising at least one composition disclosed in the present invention. | 01-13-2011 |
20110046360 | ENA NUCLEIC ACID DRUGS MODIFYING SPLICING IN mRNA PRECURSOR - Oligonucleotides having a nucleotide sequence complementary to nucleotide numbers such as 2571-2607, 2578-2592, 2571-2592, 2573-2592, 2578-2596, 2578-2601 or 2575-2592 of the dystrophin cDNA (Gene Bank accession No. NM_004006.1) and therapeutic agents for muscular dystrophy comprising such oligonucleotides. | 02-24-2011 |
20110054159 | RNA INTERFERENCE MEDIATING SMALL RNA MOLECULES - Double-stranded RNA (dsRNA) induces sequence-specific post-transcriptional gene silencing in many organisms by a process known as RNA interference (RNAi). Using a | 03-03-2011 |
20110054160 | THERAPEUTIC COMPOSITIONS - This application relates to therapeutic siRNA agents and methods of making and using the agents. | 03-03-2011 |
20110065908 | METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION BY DOUBLE-STRANDED RNA - The invention provides compositions and methods for selectively reducing the expression of a gene product from a desired target gene, as well as treating diseases caused by expression of the gene. The method involves introducing into the environment of a cell an amount of a double-stranded RNA (dsRNA) such that a sufficient portion of the dsRNA can enter the cytoplasm of the cell to cause a reduction in the expression of the target gene. The dsRNA has a first oligonucleotide sequence that is between 26 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of from about 19 to about 23 nucleotides is complementary to a nucleotide sequence of the RNA produced from the target gene. | 03-17-2011 |
20110087015 | Nucleoside and nucleotide having an unnatural base and use thereof - The object of the present invention is to provide a nucleoside or a nucleotide, or a derivative thereof, which has an unnatural base. The nucleoside and others of the present invention are characterized by having a 2-amino-6-(2-thiazolyl)purin-9-yl group or a 2-amino-6-(2-oxazolyl)purin-9-yl group as a base, wherein the 4- and/or 5-position of the thiazolyl or oxazolyl group may be substituted. | 04-14-2011 |
20110098460 | SiRNA Useful to Suppress expression of eIF-5A1 - The present invention relates to apoptosis specific eucaryotic initiation factor 5A (eIF-5A), referred to as apoptosis factor 5A1 or simply factor 5A1, apoptosis factor 5A1 nucleic acids and polypeptides and methods for inhibiting or suppressing apoptosis in cells using antisense nucleotides or siRNAs to inhibit expression of factor 5A1. The invention also relates to suppressing or inhibiting expression of pro-inflammatory cytokines by inhibiting expression of apoptosis factor 5A. | 04-28-2011 |
20110098461 | Novel RNA Interference Methods Using DNA-RNA Duplex Constructs - The present invention provides novel compositions and methods for suppressing the function or activity of a targeted gene through a novel intracellular piRNA-mediated RNAi mechanism, using RNA-DNA duplex constructs. The invention further provides novel methods and compositions for generating or producing RNA-DNA duplex agents, whose quantity is high enough to be used for the invention's gene silencing transfection and possibly in therapeutics applications. This improved RNA-polymerase chain reaction (RNA-PCR) method utilizes thermocycling steps of promoter-linked DNA or RNA template synthesis, in vitro transcription and then reverse transcription to bring up the amount of RNA-DNA duplexes up to two thousand folds within one round of the above procedure for using in D-RNAi-directed gene silencing. | 04-28-2011 |
20110112283 | RNA INTERFERENCE MEDIATING SMALL RNA MOLECULES - Double-stranded RNA (dsRNA) induces sequence-specific post-transcriptional gene silencing in many organisms by a process known as RNA interference (RNAi). Using a | 05-12-2011 |
20110112284 | METHODS FOR MODULATING IKKALPHA ACTIVITY - A method for modulating NF-κB dependent gene transcription in a cell comprised of modulating IKKα protein activity in the cell. The present invention also provides siRNA compositions and methods thereof for modulating NF-κB dependent gene transcription. | 05-12-2011 |
20110118456 | Interfering RNA Molecules - The present invention is related to a ribonucleic acid comprising a double stranded structure whereby the double-stranded structure comprises a first strand and a second strand, whereby the first strand comprises a first stretch of contiguous nucleotides and whereby said first stretch is at least partially complementary to a target nucleic acid, and the second strand comprises a second stretch of contiguous nucleotides whereby said second stretch is at least partially identical to a target nucleic acid, and whereby the double stranded structure is blunt ended. | 05-19-2011 |
20110124853 | Conjugates and Compositions for Cellular Delivery - This invention features conjugates, degradable linkers, compositions, methods of synthesis, and applications thereof, including cholesterol, folate, galactose, galactosamine, N-acetyl galactosamine, PEG, phospholipid, peptide and human serum albumin (HSA) derived conjugates of biologically active compounds, including antibodies, antivirals, chemotherapeutics, peptides, proteins, hormones, nucleosides, nucleotides, non-nucleosides, and nucleic acids including enzymatic nucleic acids, DNAzymes, allozymes, antisense, dsRNA, siNA, siRNA, triplex oligonucleotides, 2,5-A chimeras, decoys and aptamers. | 05-26-2011 |
20110130557 | INTERCALATING TRIPLEXES AND DUPLEXES USING ARYL NAPHTHOIMIDAZOL AND PROCESS FOR THE PREPARATION THEREOF - There is provided an intercalating oligonucleotide for stabilizing natural or modified DNA and RNA triplexes, duplexes and hybrids thereof having the general structure (I) triplex forming oligonucleotides of the invention are capable of binding specifically to double stranded target nucleic acids and are therefore of interest for modulation of the activity of target nucleic acids and also detection of target nucleic acids. | 06-02-2011 |
20110160445 | Heparin-Binding Growth Factor (HBGF) Polypeptides - Substantially pure heparin-binding growth factor polypeptides (HBGFs), nucleic acids encoding the HBGFs and antibodies which bind to the HBGFs of the invention are provided. The HBGF polypeptides axe useful in methods for the induction of bone, cartilage and tissue formation, growth and development of the endometrium and in the acceleration of wound healing. | 06-30-2011 |
20110178283 | Ribonucleic acid interference molecules and binding sites derived by analyzing intergenic and intronic regions of genomes - Sequences that can be used in the context of controlled gene regulation are provided. In one aspect, at least one sequence comprising at least one of one or more sequences having SEQ ID NO: 1 through SEQ ID NO: 747,326 is provided. One or more of the provided sequences may be computationally predicted, e.g., from publicly available genomes, using a method based on pattern discovery. In another aspect, a method for regulating the expression of a transcript comprises the step of said transcript containing a region that corresponds to at least one of the provided sequences having SEQ ID NO: 1 through SEQ ID NO: 747,326, the region being targeted either by a naturally occurring, or appropriately designed, interfering RNA molecule that regulates the expression of said transcript through post-transcriptional silencing. In a third aspect, a method for regulating the expression of a transcript comprises the step of at least one of the provided sequences having SEQ ID NO: 1 through SEQ ID NO: 747,326 being used to design an interfering RNA molecule that contains a region that corresponds to the reverse complement of one or more of the one or more sequences having SEQ ID NO: 1 through SEQ ID NO: 747,326, the interfering molecule regulating, through post-transcriptional silencing, one or more transcripts that contain said sequence of the one or more sequences having SEQ ID NO: 1 through SEQ ID NO: 747,326, or a substantial fraction thereof. | 07-21-2011 |
20110201798 | THERAPEUTIC COMPOSITIONS - This application relates to therapeutic siRNA agents and methods of making and using the agents. | 08-18-2011 |
20110213136 | ANTISENSE MODULATION OF STEAROYL-COA DESATURASE EXPRESSION - Antisense compounds, compositions and methods are provided for modulating the expression of stearoyl-CoA desaturase. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding stearoyl-CoA desaturase. Methods of using these compounds for modulation of stearoyl-CoA desaturase expression and for treatment of diseases associated with expression of stearoyl-CoA desaturase are provided. | 09-01-2011 |
20110218334 | PHOSPHOROTHIOATE OLIGONUCLEOTIDES AND NON-NUCLEOSIDIC PHOSPHOROTHIOATES AS DELIVERY AGENTS FOR iRNA AGENTS - The invention relates to use of single-stranded phosphorothioate oligonucleotides or non-nucleosidic phosphrothiaote as vehicles or carriers to modulate the biodistribution of iRNA agents. | 09-08-2011 |
20110224418 | MODIFIED sIRNA MOLECULES AND USES THEREOF - The present invention provides chemically modified siRNA molecules and methods of using such siRNA molecules to silence target gene expression. Advantageously, the modified siRNA of the present invention is less immunostimulatory than its corresponding unmodified siRNA sequence and retains full RNAi activity against the target sequence. The present invention also provides nucleic acid-lipid particles comprising a modified siRNA, a cationic lipid, and a non-cationic lipid, which can further comprise a conjugated lipid that inhibits aggregation of particles. Methods for identifying and/or modifying an siRNA having immunostimulatory properties are also provided. | 09-15-2011 |
20110245481 | METHOD FOR INHIBITING FUNCTION OF micro-RNA - A miRNA-inhibiting RNA complex has a double-stranded structure, in which at least one RNA strand that includes a miRNA-binding sequence is linked to the two strands at at least one end of the double-stranded structure. The complex can efficiently inhibit miRNAs. In particular, RNAs in which two RNAs containing a miRNA binding sequence are positioned between two double-stranded structures were able to strongly inhibit miRNA. These RNAs can be expressed from, for example, a PolIII promoter, and by integration into a vector, miRNAs can be stably inhibited for a long period of time. | 10-06-2011 |
20110257384 | METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION BY DOUBLE-STRANDED RNA - The invention is directed to compositions and methods for selectively reducing the expression of a gene product from a desired target gene in a cell, as well as for treating diseases caused by the expression of the gene. More particularly, the invention is directed to compositions that contain double stranded RNA (“dsRNA”), and methods for preparing them, that are capable of reducing the expression of target genes in eukaryotic cells. The dsRNA has a first oligonucleotide sequence that is between 25 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of at least 19 nucleotides is sufficiently complementary to a nucleotide sequence of the RNA produced from the target gene to trigger the destruction of the target RNA by the RNAi machinery. | 10-20-2011 |
20110269951 | siRNA targeting cyclin-dependent kinase inhibitor 1B (p27, Kip1) (CDKN1B) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for CDKN1B. | 11-03-2011 |
20110313144 | iRNA Agents Targeting CCR5 Expressing Cells And Uses Thereof - The invention relates to iRNA agents that preferably include a modification that targets CC chemokine receptor 5 (CCR5). The invention also relates to methods of making and using such modified iRNA agents. | 12-22-2011 |
20120004403 | RNA INTERFERENCE MEDIATED INHIBITION OF TNF AND TNF RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating tumor necrosis factor and/or tumor necrosis factor receptor gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of tumor necrosis factor and/or tumor necrosis factor receptor gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of tumor necrosis factor and/or tumor necrosis factor receptor genes, (TNF and/or TNF receptor). | 01-05-2012 |
20120022245 | FOLATE TARGETING OF NUCLEOTIDES - The present invention relates to compounds, compositions, kits, and methods of use in targeting nucleotides, such as siRNA's, to cancer cells or to immune system cells involved in inflammation. More particularly, the invention is directed to receptor binding ligand-nucleotide delivery conjugates for use in specifically targeting the conjugates to cancer cells or to immune system cells, methods of treatment with these conjugates, methods of preparation of these conjugates, and methods of reducing the expression of a gene in vitro or in vivo with the conjugates described herein. | 01-26-2012 |
20120041184 | RNA INTERFERENCE MEDIATED INHIBITION OF HUMAN IMMUNODEFICIENCY VIRUS (HIV) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - This invention relates to compounds, compositions, and methods useful for modulating human immunodeficiency virus (HIV) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of human immunodeficiency virus (HIV) gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of HIV genes. The small nucleic acid molecules are useful in the treatment of HIV infection, AIDS, and/or diseases and conditions related to HIV infection and/or AIDS in a subject or organism. | 02-16-2012 |
20120059159 | DIFFERENTIATION THERAPY FOR SARCOMAS - Use of muscle-enriched/specific microRNAs for the treatment of sarcomas, as for example rhabdomyosarcomas, synovial sarcoma, alveolar soft part sarcoma, liposarcoma, and osteosarcoma, wherein the microRNAs are able to stop the development of neoplastic cells and to force the neoplastic cells to differentiate into terminally differentiated muscle cells. | 03-08-2012 |
20120071641 | NUCLEIC ACID CHEMICAL MODIFICATIONS - The present invention provides nucleosides of formula (1) and oligonucleotides comprising at least one nucleoside of formula (2): Another aspect of the invention relates to a method of inhibiting the expression of a gene in cell, the method comprising (a) contacting an oligonucleotide of the invention with the cell; and (b) maintaining the cell from step (a) for a time sufficient to obtain degradation of the mRNA of the target gene. | 03-22-2012 |
20120083596 | Pharmaceutical Composition - The invention provides pharmaceutical compositions comprising short single stranded oligonucleotides, of length of between 8 and 17 nucleobases which are complementary to human microRNAs. The short oligonucleotides are particularly effective at alleviating miRNA repression in vivo. It is found that the incorporation of high affinity nucleotide analogues into the oligonucleotides results in highly effective anti-microRNA molecules which appear to function via the formation of almost irreversible duplexes with the miRNA target, rather than RNA cleavage based mechanisms, such as mechanisms associated with RNaseH or RISC. | 04-05-2012 |
20120088907 | OLIGOMERIC COMPOUNDS FOR THE MODULATION OF SURVIVIN EXPRESSION - Oligonucleotides directed against the survivin gene are provided for modulating the expression of survivin. The compositions comprise oligonucleotides, particularly antisense oligonucleotides, targeted to nucleic acids encoding the survivin. Methods of using these compounds for modulation of survivin expression and for the treatment of diseases associated with either overexpression of survivin, expression of mutated survivin or both are provided. Examples of diseases are cancer such as lung, breast, colon, prostate, pancreas, lung, liver, thyroid, kidney, brain, testes, stomach, intestine, bowel, spinal cord, sinuses, bladder, urinary tract or ovaries cancers. The oligonucleotides may be composed of deoxyribonucleosides or a nucleic acid analogue such as for example locked nucleic acid or a combination thereof. | 04-12-2012 |
20120108803 | SIRNA CONJUGATE AND PREPARATION METHOD THEREOF - Provided are an siRNA-polymer conjugate, and a method for preparing the same, and more specifically, to a hybrid conjugate formed by covalently bonding siRNA and a polymeric compound for improving the in vivo stability of siRNA, and to a preparation method of the hybrid conjugate. The conjugate of the present invention can improve the in vivo stability of siRNA, thereby achieving an efficient delivery of therapeutic siRNA into cells and exhibiting the activity of siRNA even with a small dose of a relative low concentration. Therefore, the conjugate can advantageously be used as not only an siRNA treatment tool for cancers and other infectious disease, but also a novel type siRNA delivery system. | 05-03-2012 |
20120130060 | Modified antisense oligopeptides with anticancer properties and method of obtaining them - This invention may be used in human and veterinary medicine for the creation of a drug that is effective in the treatment of oncological illnesses in animals and humans. | 05-24-2012 |
20120136144 | SIRNA TARGETING CATENIN, BETA-1 (CTNNB1) - Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for CTNNB1. | 05-31-2012 |
20120136145 | Compositions and Methods for Inhibiting Expression of Eg5 Gene - The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Eg5 gene (Eg5 gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of the Eg5 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by Eg5 expression and the expression of the Eg5 gene using the pharmaceutical composition; and methods for inhibiting the expression of the Eg5 gene in a cell. | 05-31-2012 |
20120149893 | MODULATION OF EIF4E-BP2 - Compounds, compositions and methods are provided for modulating the expression of eIF4E-BP2. The compositions comprise oligonucleotides, targeted to nucleic acid encoding eIF4E-BP2. Methods of using these compounds for modulation of eIF4E-BP2 expression and for diagnosis and treatment of diseases and conditions associated with expression of eIF4E-BP2 are provided. | 06-14-2012 |
20120202982 | Compositions and Methods for Therapy of Macular Degeneration - Provided are compositions and methods for therapy of macular degeneration including dry age-related macular degeneration (dAMD), juvenile macular degenerations (JMDs) where toxic retinoids are known to accumulate as part of the pathogenesis, such as Stargardt disease, and Best disease, and neovascular wet age-related macular degeneration. The method entails administering to an individual in need of therapy for macular degeneration a first polynucleotide that can facilitate a reduction in the amount of rod opsin (RHO) mRNA in the individual; or a second polynucleotide that can facilitate a reduction in the amount of RPE65 mRNA in the individual; or a combination thereof. The polynucleotides of the invention are hammerhead ribozymes or shRNAs. The polynucleotides target a sequence in RHO mRNA or RPE65 mRNA and facilitate reduction in the target mRNA via ribozymatic cleavage of the target, or by hybridization to the target, which leads to RNAi mediated degradation of the target mRNA. | 08-09-2012 |
20120232260 | AMPHIPHILIC NUCLEOTIDE COCHLEATE COMPOSITIONS AND METHODS OF USING THE SAME - The present invention is directed to cochleate compositions that include a nucleotide that is associated with a positively charged amphiphile. The present invention also includes methods for making and using the compositions provided herein. | 09-13-2012 |
20120283424 | REDUCTION OF OFF-TARGET RNA INTERFERENCE TOXICITY - The present invention is directed to RNA interference (RNAi) molecules targeted against a nucleic acid sequence, and methods of using these RNAi molecules to reduce off-target toxicity. | 11-08-2012 |
20130066058 | Use of Antisense Oligonucleotides or siRNA to Suppress Expression of eIF-5A1 - The present invention relates to apoptosis specific eucaryotic initiation factor 5A (eIF-5A), referred to as apoptosis factor 5A1 or simply factor 5A1, apoptosis factor 5A1 nucleic acids and polypeptides and methods for inhibiting or suppressing apoptosis in cells using antisense nucleotides or siRNAs to inhibit expression of factor 5A1. The invention also relates to suppressing or inhibiting expression of pro-inflammatory cytokines by inhibiting expression of apoptosis factor 5A. | 03-14-2013 |
20130066059 | CYCLIZING LINKER AND CYCLIC CAGED MORPHOLINOS - A bifunctional and photocleavable linker for cyclizing a morpholino-based oligonucleotide, having the formula: | 03-14-2013 |
20130072671 | MODULATION OF EXON RECOGNITION IN PRE-MRNA BY INTERFERING WITH THE SECONDARY RNA STRUCTURE - The invention provides a method for generating an oligonucleotide with which an exon may be skipped in a pre-mRNA and thus excluded from a produced mRNA thereof. Further provided are methods for altering the secondary structure of an mRNA to interfere with splicing processes and uses of the oligonucleotides and methods in the treatment of disease. Further provided are pharmaceutical compositions and methods and means for inducing skipping of several exons in a pre-mRNA. | 03-21-2013 |
20130079505 | BIVALENT ANTISENSE OLIGONUCLEOTIDES - The present invention provides bivalent molecules comprising a first oligonucleotide linked to a second oligonucleotide. The first and the second oligonucleotide are preferably linked via a linking moiety. Preferably, both the first and/or the second oligonucleotide comprise an antisense sequence complementary to a cellular RNA such as mRNA or microRNA. | 03-28-2013 |
20130090465 | ENA NUCLEIC ACID PHARMACEUTICALS CAPABLE OF MODIFYING SPLICING OF mRNA PRECURSORS - Oligonucleotides having a nucleotide sequence complementary to nucleotide numbers such as 2571-2607, 2578-2592, 2571-2592, 2573-2592, 2578-2596, 2578-2601 or 2575-2592 of the dystrophin cDNA (Gene Bank accession No. NM_004006.1) and therapeutic agents for muscular dystrophy comprising such oligonucleotides. | 04-11-2013 |
20130096288 | siRNA conjugate and preparation method thereof - Provided are an siRNA-polymer conjugate, and a method for preparing the same, and more specifically, to a hybrid conjugate formed by covalently bonding siRNA and a polymeric compound for improving the in vivo stability of siRNA, and to a preparation method of the hybrid conjugate. The conjugate of the present invention can improve the in vivo stability of siRNA, thereby achieving an efficient delivery of therapeutic siRNA into cells and exhibiting the activity of siRNA even with a small dose of a relative low concentration. Therefore, the conjugate can advantageously be used as not only an siRNA treatment tool for cancers and other infectious disease, but also a novel type siRNA delivery system. | 04-18-2013 |
20130096289 | HYDROXYMETHYL SUBSTITUTED RNA OLIGONUCLEOTIDES AND RNA COMPLEXES - The present invention is directed to RNA oligonucleotides or complexes of RNA oligonucleotides, denoted herein together as RNA complexes, containing at least one, but optionally more that one, hydroxymethyl substituted nucleotide monomer(s). By a hydroxymethyl substituted nucleotide monomer is understood a nucleotide monomer containing a hydroxymethyl group (that may be unsubstituted, O-substituted for example with a conjugating group, or converted into an optionally substituted or conjugated aminomethyl group). This hydroxymethyl group is not partaking in formation of an internucleotide linkage and is not the hydroxymethyl group (containing the 5′-hydroxy group) of a natural RNA monomer. The RNA complexes of the invention may be useful for therapeutic applications, diagnostic applications or research applications. | 04-18-2013 |
20130096290 | SINGLE STRANDED EXTENDED DICER SUBSTRATE AGENTS AND METHODS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION - The invention provides compositions and methods for reducing expression of a target gene in a cell, involving contacting a cell with an isolated double stranded nucleic acid (dsNA) in an amount effective to reduce expression of a target gene in a cell. The dsNAs of the invention possess a single stranded extension (in most embodiments, the single stranded extension comprises at least one modified nucleotide and/or phosphate back bone modification). Such single stranded extended Dicer-substrate siRNAs (DsiRNAs) were demonstrated to be effective RNA inhibitory agents compared to corresponding double stranded DsiRNAs. | 04-18-2013 |
20130102768 | EFFICIENT METHOD FOR NUCLEAR REPROGRAMMING - A method of preparing induced pluripotent stem cells, comprising a nuclear reprogramming step with a nuclear reprogramming factor in the presence of miRNA, wherein said miRNA has a property of providing a higher nuclear reprogramming efficiency in the presence of said miRNA than in the absence thereof. | 04-25-2013 |
20130102769 | INTERFERING RNA MOLECULES - The present invention is related to a ribonucleic acid comprising a double stranded structure whereby the double-stranded structure comprises a first strand and a second strand, whereby the first strand comprises a first stretch of contiguous nucleotides and whereby said first stretch is at least partially complementary to a target nucleic acid, and the second strand comprises a second stretch of contiguous nucleotides whereby said second stretch is at least partially identical to a target nucleic acid, and whereby the double stranded structure is blunt ended. | 04-25-2013 |
20130109849 | COMPOSITIONS AND THEIR USES DIRECTED TO ACEYTL-COA CARBOXYLASES | 05-02-2013 |
20130109850 | MODIFIED 5' DIPHOSPHATE NUCLEOSIDES AND OLIGOMERIC COMPOUNDS PREPARED THEREFROM | 05-02-2013 |
20130123485 | CATIONIC LIPIDS, METHODS FOR PREPARING THE SAME, AND DELIVERY SYSTEMS HAVING ABILITY TO TRANSITION INTO CELLS COMPRISING THE SAME - The present invention provides cationic lipids, methods for preparing the same, and delivery systems comprising the same. The present invention can provide cationic lipids which enhance the efficiency of intracellular or in vivo delivery of multiple-anionic target compounds such as drugs, anticancer agents, nucleic acids, etc., have no intracellular toxicity, but show increased stability, methods for preparing the same, and delivery systems comprising the same. | 05-16-2013 |
20130131331 | CANCER-CELL-SPECIFIC CYTOSTATIC AGENT - The present inventors discovered that although suppressing expression of the RecQ1 gene, a RecQ helicase family gene, shows suppressive effects on cell proliferation in cancer cells, such effects are not observed in human TIG3 cells (a normal diploid fibroblast cell line), which are normal cells. Hence, the present inventors discovered that siRNAs against RecQ1 gene have cancer cell-specific cell proliferation-suppressing effects that are mediated by suppression of the expression of said gene. | 05-23-2013 |
20130144048 | THERAPEUTIC COMPOSITIONS - This application relates to therapeutic siRNA agents and methods of making and using the agents. | 06-06-2013 |
20130150569 | BICYCLIC NUCLEOSIDES AND OLIGOMERIC COMPOUNDS PREPARED THEREFROM - The present invention provides novel 3′,5′-linked bicyclic nucleosides and oligomeric compounds prepared therefrom. The bicyclic nucleosides provided herein are useful for enhancing one or more properties of the oligomeric compounds they are incorporated into such as nuclease resistance. | 06-13-2013 |
20130190484 | In Vivo Polynucleotide Delivery Conjugates Having Enzyme Sensitive Linkages - The present invention is directed compositions for delivery of RNA interference (RNAi) polynucleotides to cells in vivo. The compositions comprise amphipathic membrane active polyamines reversibly modified with enzyme cleavable dipeptide-amidobenzyl-carbonate masking agents. Modification masks membrane activity of the polymer while reversibility provides physiological responsiveness. The reversibly modified polyamines (dynamic polyconjugate or DPC) are further covalently linked to an RNAi polynucleotide or co-administered with a targeted RNAi polynucleotide-targeting molecule conjugate. | 07-25-2013 |
20130197207 | METHOD OF INHIBITING ALU RNA AND THERAPEUTIC USES THEREOF - The presently-disclosed subject matter includes methods of identifying an Alu RNA inhibitor, and methods and compositions for inhibiting Alu RNA. Methods and compositions can be used for the treatment of geographic atrophy and other conditions of interest. | 08-01-2013 |
20130203977 | OLIGONUCLEOTIDES COMPRISING ALTERNATING SEGMENTS AND USES THEREOF - The invention relates to oligonucleotides having alternating segments of sugar-modified nucleosides and 2′-deoxynucleosides, and uses thereof. The invention further related to oligonucleotides having alternating segments of sugar-modified nucleotides and 2′-deoxynucleotides, and uses thereof. Such uses include the preparation of antisense oligonucleotides and their use for the prevention or depletion of function of a target nucleic acid of interest, such as an RNA, in a system. Accordingly, and oligonucleotide of the invention is useful for therapeutic, analytical and diagnostic methods and uses, as well as component of compositions and commercial packages corresponding to such methods and uses. | 08-08-2013 |
20130211061 | Method and compositions for the identification of agents that have a potential effect against chronic inflammatory diseases - The present invention is based on two important experimental observations: The first observation is that increased extracellular concentrations of ionized calcium are found in erosive arthritis and stimulate monocytic IL-1β release via the CaSR and GPRC6A. Simultaneous stimulation of monocytes with calcium ions and selected TLR ligands results in a 20-fold increased IL1β response compared to lipopolysaccharide (LPS) alone. During the crosstalk between GPCR and TLR signaling, phospholipase C is activated, which triggers calcium dependent potassium channels, resulting in potassium efflux, caspase-1 activation and IL-1β release. The amplification of IL1β secretion at sites of locally increased calcium ion concentrations aggravates rheumatoid arthritis. The second important observation is that both CaSR and GPRC6A, are highly expressed in the synovial membrane of patients with rheumatoid arthritis, but expression of GPRC6A, but not of CaSR, is lower in patients with osteoarthritis (s. FIG. | 08-15-2013 |
20130211062 | ANTISENSE NUCLEIC ACIDS - The present invention provides a pharmaceutical composition which causes skipping of the 53rd exon in the human dystrophin gene with a high efficiency. | 08-15-2013 |
20130211063 | MODIFIED iRNA AGENTS - The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose. The inclusion of such a monomer can allow for modulation of a property of the iRNA agent into which it is incorporated, e.g., by using the non-ribose moiety as a point to which a ligand or other entity, e.g., a lipophilic moiety. e.g., cholesterol, is directly, or indirectly, tethered. The invention also relates to methods of making and using such modified iRNA agents. | 08-15-2013 |
20130211064 | TETRAHYDROPYRAN NUCLEIC ACID ANALOGS - The present disclosure describes tetrahydropyran nucleoside analogs, oligomeric compounds prepared therefrom and methods of using the oligomeric compounds. More particularly, tetrahydropyran nucleoside analogs are provided, having one or more chiral substituents, that are useful for enhancing properties of oligomeric compounds including nuclease resistance and binding affinity. In some embodiments, the oligomeric compounds provided herein hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. | 08-15-2013 |
20130253180 | ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHODS OF USE THEREOF - Antisense molecules capable of binding to a selected target site in the dystrophin gene to induce exon skipping are described. | 09-26-2013 |
20130281684 | COMPOSITIONS AND THEIR USES DIRECTED TO HUNTINGTIN - Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal. Further provided are methods of slowing or preventing Huntington's Disease (HD) progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntington's Disease (HD) in an individual susceptible to Huntington's Disease (HD). Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. | 10-24-2013 |
20130281685 | iRNA AGENTS WITH BIOCLEAVABLE TETHERS - The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose that further includes a tether having one or more linking groups, in which at least one of the linking groups is a cleavable linking group. The tether in turn can be connected to a selected moiety, e.g., a ligand, e.g., a targeting or delivery moiety, or a moiety which alters a physical property. The cleavable linking group is one which is sufficiently stable outside the cell such that it allows targeting of a therapeutically beneficial amount of an iRNA agent (e.g., a single stranded or double stranded iRNA agent), coupled by way of the cleavable linking group to a targeting agent—to targets cells, but which upon entry into a target cell is cleaved to release the iRNA agent from the targeting agent. | 10-24-2013 |
20140005374 | CHIMERIC OLIGOMERIC COMPOUNDS FOR MODULATION OF SPLICING | 01-02-2014 |
20140018527 | METHODS AND COMPOSITIONS FOR THE TREATMENT OF EYE DISORDERS WITH INCREASED INTRAOCULAR PRESSURE - The present invention relates to methods and compositions that decrease intraocular pressure (IOP) of the eye. The compositions of the invention comprise short interfering nucleic acid molecules (siNA) including, but not limited to, siRNA that decrease expression of genes associated with production or drainage of intraocular fluid. The compositions of the invention can be used in the preparation of a medicament for the treatment of eye conditions displaying increased IOP such as glaucoma, infection, inflammation, uveitis, and diabetic retinopathy. The methods of the invention comprise the administration to a patient in need thereof of an effective amount of one or more siNAs of the invention. | 01-16-2014 |
20140024821 | OLIGONUCLEOTIDE ANALOGUES TARGETING HUMAN LMNA - Provided are LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin. | 01-23-2014 |
20140051844 | Nucleic Acid Purification - Methods and composition for nucleic acid isolation are provided. In one embodiment, a method is provided for nucleic acid purification from biological samples, such as whole blood samples, extracted with phenol-based denaturing solvents, which does not require phase separation or nucleic acid precipitation. Methods according to the invention may also be used for of small RNAs (e.g., siRNAs or miRNAs) purification and are amenable to automation. | 02-20-2014 |
20140081012 | NANOPARTICLE, LIPOSOMES, POLYMERS, AGENTS AND PROTEINS MODIFIED WITH REVERSIBLE LINKERS - Pharmaceutical, chemical and biological agents containing a reversible disulfide linker are described. These agents can also be covalently bound or contained in delivery vehicles for delivering the agents to desired targets or areas. Also described are delivery vehicles which contain an agent having a reversible disulfide linker and to vehicles that are covalently linked to the agent containing a reversible disulfide linker. The modifications described herein can modify properties of the agents and vehicles, thereby providing desired solubility, stability, hydrophobicity and targeting while the reversibility of the linker can leave the agent to which it is attached free from residual chemical groups after being reduced. | 03-20-2014 |
20140088300 | Modified siRNA Molecules Incorporating 5-Fluoro-2'-Deoxyuridine Residues to Enhance Cytotoxicity - A synthesized siRNA molecule having the sense strand with one or more uridine bases replaced by one or more respective nucleoside analogs, such as 5-fluoro-2′-deoxyuridine (FdU). | 03-27-2014 |
20140107330 | OLIGOMERIC COMPOUNDS COMPRISING BICYCLIC NUCLEOTIDES AND USES THEREOF - The present invention provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell. | 04-17-2014 |
20140114057 | ANTISENSE COMPOUNDS TARGETING GENES ASSOCIATED WITH USHER SYNDROME - The present invention provides compounds comprising oligonucleotides complementary to an Usher transcript. Certain such compounds are useful for hybridizing to an Usher transcript, including but not limited, to an Usher transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the Usher transcript. In certain such embodiments, the Usher transcript includes a mutation that results in cryptic splicing and hybridization of the oligonucleotide results in a decrease in the amount of cryptic splicing. In certain embodiments, such compounds are used to treat Usher Syndrome. | 04-24-2014 |
20140121365 | OLIGOMERIC COMPOUNDS AND COMPOSITIONS FOR USE IN MODULATION OF SMALL NON-CODING RNAS - Compounds, compositions and methods are provided for modulating the expression and function of small non-coding RNAs. The compositions comprise oligomeric compounds, targeted to small non-coding RNAs. Methods of using these compounds for modulation of small non-coding RNAs as well as downstream targets of these RNAs and for diagnosis and treatment of disease associated with small non-coding RNAs are also provided. | 05-01-2014 |
20140128591 | ANTISENSE DESIGN - A novel class of pharmaceuticals which comprises a Locked Nucleic Acid (LNA) which can be used in antisense therapy. These novel oligonucleotides have improved antisense properties. The novel oligonucleotides are composed of at least one LNA selected from beta-D-thio/amino-LNA or alpha-L-oxy/thio/amino-LNA. The oligonucleotides comprising LNA may also include DNA and/or RNA nucleotides. | 05-08-2014 |
20140128592 | METHODS AND MEANS FOR EFFICIENT SKIPPING OF EXON 45 IN DUCHENNE MUSCULAR DYSTROPHY PRE-mRNA - The invention relates to a method for inducing or promoting skipping of exon 45 of DMD pre-mRNA in a Duchenne Muscular Dystrophy patient, preferably in an isolated (muscle) cell, the method comprising providing an isolate muscle cell with a molecule that binds to a continuous stretch of at least 21 nucleotides within said exon. The invention further relates to such molecule used in the method. | 05-08-2014 |
20140135488 | BRACHYURY POLYPEPTIDES AND METHODS FOR USE - It is disclosed herein that Brachyury is expressed in human tumors, specifically in tumors of the small intestine, stomach, kidney, bladder, uterus, ovary, and testes, as well as in lung, colon and prostate carcinomas. Immunogenic Brachyury polypeptides are disclosed herein. These polypeptides can be used in diagnostic assays for Brachyury expression, as well as for inducing an immune response to Brachyury. Polynucleotides encoding the immunogenic Brachyury polypeptides, vectors including these polypeptides, host cells transformed with these vectors, and methods of using these polypeptides, polynucleotides, vectors, and host cells are provided. Methods of diagnosing a Brachyury-expressing cancer are also provided. Exemplary cancers include small lung, colon, intestine, stomach, kidney, bladder, uterus, ovary, and testes and prostate cancers. Methods of treating cancer are also disclosed. | 05-15-2014 |
20140155587 | ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHODS OF USE THEREOF - An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 202. | 06-05-2014 |
20140163214 | RNA INTERFERENCE SUPPRESSION OF NEURODEGENERATIVE DISEASES AND METHODS OF USE THEREOF - The present invention is directed to RNA interference (RNAi) molecules targeted against a nucleic acid sequence that encodes poly-glutamine repeat diseases, and methods of using these RNAi molecules. | 06-12-2014 |
20140194612 | IMMUNOSUPPRESSION COMPOUND AND TREATMENT METHOD - A method and compound for suppressing an immune response in a mammalian subject, for the treatment or prevention of an autoimmune condition or transplantation rejection are disclosed. The compound is an antisense oligonucleotide analog compound having a targeting sequence complementary to a preprocessed CTLA-4 mRNA region identified by SEQ ID NO: 22 in SEQ ID NO: 1, spanning the splice junction between intron 1 and exon 2 of the preprocessed mRNA of the subject. The compound is effective, when administered to a subject, to form within host cells, a heteroduplex structure (i) composed of the preprocessed CTLA-4 mRNA and the oligonucleotide compound, (ii) characterized by a Tm of dissociation of at least 45° C., and (iii) resulting in an increased ratio of processed mRNA encoding ligand-independent CTLA-4 to processed mRNA encoding full-length CTLA-4. | 07-10-2014 |
20140206854 | TARGETING MICRORNAS FOR THE TREATMENT OF LIVER CANCER - Provided herein are methods for the treatment of liver cancer. These methods encompass the administration of a compound comprising a modified oligonucleotide, wherein the modified oligonucleotide is targeted to a miRNA. Also provided herein are compositions for the treatment of liver cancer. Such compositions include compounds comprising a modified oligonucleotide, wherein the modified oligonucleotide is targeted to a miRNA. Certain miRNAs have been identified as overexpressed in liver cancer, such as, for example, hepatocellular carcinoma, and are thus selected for targeting by modified oligonucleotides. Further, certain miRNAs have been identified as overexpressed in hepatocellular carcinoma cells exposed to dioxin, and are thus selected for targeting by modified oligonucleotides. Antisense inhibition of certain of these miRNAs has been found to inhibit cell proliferation and induce apoptosis. | 07-24-2014 |
20140235844 | Short Interfering RNA (siRNA) Analogues - The present invention is directed to novel double-stranded short interfering (siRNA) analogues comprising locked nucleic acid (LNA) monomers. Such compounds induces sequence-specific post-transcriptional gene silencing in many organisms by a process known as RNA interference (RNAi). The compounds disclosed herein has improved properties compared to non-modified siRNAs and may, accordingly, be useful as therapeutic agents, e.g., in the treatment of various cancer forms. More particularly, the present invention is directed to siRNA analogues comprising a sense strand and an antisense strand, wherein each strand comprises 12-35 nucleotides and wherein the siRNA analogues comprise at least one locked nucleic acid (LNA) monomer. | 08-21-2014 |
20140243515 | ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHODS OF USE THEREOF - An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 202. | 08-28-2014 |
20140243516 | ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHODS OF USE THEREOF - An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 202. | 08-28-2014 |
20140249304 | LONG INTERFERING DSRNA SIMULTANEOUSLY INDUCING AN IMMUNE REACTION AND THE INHIBITION OF THE EXPRESSION OF TARGET GENES - A long interfering dsRNA (liRNA) capable of inhibiting specific RNAi-mediated expression of target genes and promoting an immune reaction, and use thereof are provided. The long interfering dsRNA can be useful in inhibiting specific expression of target genes through an RNA-interfering reaction in a sequence-specific manner and inducing expression of interferon-β by stimulating a protein kinase R (PKR) path in a structure-dependent manner. | 09-04-2014 |
20140288290 | ANTISENSE DESIGN - A novel class of pharmaceuticals which comprises a Locked Nucleic Acid (LNA) which can be used in antisense therapy. These novel oligonucleotides have improved antisense properties. The novel oligonucleotides are composed of at least one LNA selected from beta-D-thio/amino-LNA or alpha-L-oxy/thio/amino-LNA. The oligonucleotides comprising LNA may also include DNA and/or RNA nucleotides. | 09-25-2014 |
20140288291 | COMPOUNDS AND METHODS FOR MODULATING PROTEIN EXPRESSION - The present invention provides compounds and methods for modulating expression of a protein, including, but not limited to, modulating splicing of a pre-mRNA to modulate the amount of one or more variants of a protein. | 09-25-2014 |
20140288292 | METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF BETA-CATENIN BY DOUBLE-STRANDED RNA - This invention relates to compounds, compositions, and methods useful for reducing β-catenin target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents. | 09-25-2014 |
20140296502 | ANTISENSE DESIGN - A novel class of pharmaceuticals which comprises a Locked Nucleic Acid (LNA) which can be used in antisense therapy. These novel oligonucleotides have improved antisense properties. The novel oligonucleotides are composed of at least one LNA selected from beta-D-thio/amino-LNA or alpha-L-oxy/thio/amino-LNA. The oligonucleotides comprising LNA may also include DNA and/or RNA nucleotides. | 10-02-2014 |
20140296503 | ISOLATED dsRNA MOLECULES AND METHODS OF USING SAME FOR SILENCING TARGET MOLECULES OF INTEREST - An isolated dsRNA molecule comprising an antisense RNA sequence for regulating a target gene of interest in a plant or a phytopathogen of the plant, wherein the dsRNA sequence is flanked by two complementary sites to an smRNA or smRNAs expressed in the plant and wherein the dsRNA molecule further comprises a helicase binding site positioned so as to allow unwinding of the strands of the isolated dsRNA molecule to single stranded RNA (ssRNA) and recruitment of an RNA-dependent RNA polymerase so as to amplify the dsRNA molecule in the plant cell and generate secondary siRNA products of the dsRNA sequence. | 10-02-2014 |
20140303362 | REDUCTION OF OFF-TARGET RNA INTERFERENCE TOXICITY - The present invention is directed to RNA interference (RNAi) molecules targeted against a nucleic acid sequence, and methods of using these RNAi molecules to reduce off-target toxicity. | 10-09-2014 |
20140309411 | THERAPEUTIC COMPOSITIONS - This application relates to therapeutic siRNA agents and methods of making and using the agents. | 10-16-2014 |
20140316123 | ENA NUCLEIC ACID PHARMACEUTICALS CAPABLE OF MODIFYING SPLICING OF mRNA PRECURSORS - Oligonucleotides having a nucleotide sequence complementary to nucleotide numbers such as 2571-2607, 2578-2592, 2571-2592, 2573-2592, 2578-2596, 2578-2601 or 2575-2592 of the dystrophin cDNA (Gene Bank accession No. NM_004006.1) and therapeutic agents for muscular dystrophy comprising such oligonucleotides. | 10-23-2014 |
20140316124 | MODIDIFED iRNA AGENTS - The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose. The inclusion of such a monomer can allow for modulation of a property of the iRNA agent into which it is incorporated, e.g., by using the non-ribose moiety as a point to which a ligand or other entity, e.g., a lipophilic moiety. e.g., cholesterol, is directly, or indirectly, tethered. The invention also relates to methods of making and using such modified iRNA agents. | 10-23-2014 |
20140323707 | SELECTIVE ANTISENSE COMPOUNDS AND USES THEREOF - The present invention provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell. | 10-30-2014 |
20140323708 | SOLUBLE ENDOGLIN AND USES THEREOF - The invention provides isolated soluble endoglin polypeptides, nucleic acids encoding soluble endoglin polypeptides, antibodies that specifically bind soluble endoglin polypeptides, and kits containing these materials. The invention also provides methods for treating or decreasing the likelihood of developing a soluble endoglin-mediated disorder in a subject requiring the administration of an agent capable of reducing the expression or biological activity of a soluble endoglin polypeptide and methods for treating or decreasing the likelihood of developing a soluble endoglin-preventive disorder in a subject requiring the administration of a soluble endoglin polypeptide or a nucleic acid encoding the soluble endoglin polypeptide. The invention further provides methods for the diagnosis of a soluble endoglin-mediated disorder or a soluble endoglin-preventive disorder and methods for identifying a compound to treat a soluble endoglin-mediated or a soluble endoglin-preventive disorder. | 10-30-2014 |
20140323709 | OLIGONUCLEOTIDE AND THERAPEUTIC AGENT FOR HYPERLIPIDEMIA CONTAINING SAME AS ACTIVE INGREDIENT - The oligonucleotide of the present invention includes a sugar-modified nucleoside, the sugar-modified nucleoside has a cross-linked structure between 4′-position and 2′-position, and the oligonucleotide is capable of binding to the apolipoprotein C-III gene. According to the present invention, an oligonucleotide useful as a therapeutic agent for hyperlipidemia that is excellent in binding affinity to the apolipoprotein C-III gene, stability and safety is provided. | 10-30-2014 |
20140330004 | OLIGONUCLEOTIDE - An oligonucleotide, which has a nucleotide residue or a nucleoside residue represented by formula (I) at the 5′ end thereof, wherein the nucleotide residue or the nucleoside residue binds to an adjacent nucleotide residue via the oxygen atom at position 3, or the like; | 11-06-2014 |
20140336370 | OLIGOMERIC COMPOUNDS AND COMPOSITIONS FOR USE IN MODULATION OF SMALL NON-CODING RNAS - Compounds, compositions and methods are provided for modulating the expression and function of small non-coding RNAs. The compositions comprise oligomeric compounds, targeted to small non-coding RNAs. Methods of using these compounds for modulation of small non-coding RNAs as well as downstream targets of these RNAs and for diagnosis and treatment of disease associated with small non-coding RNAs are also provided. | 11-13-2014 |
20140336371 | SIRNA MOLECULE FOR INHIBITING GROWTH OF MELANIN AND APPLICATION THEREOF - Provided is a double-chain siRNA molecule targeting a microphthalmia-associated transcription factor MITF coding gene. A sense strand of the siRNA molecule has a sequence of SEQ ID NO: 3 and an anti-sense strand has a sequence of SEQ ID NO: 4, and the anti-sense strand specifically binds to mRNA of the MITF coding gene, to degrade the mRNA, thereby reducing the systhesis of melanin. Further provided is an application of the siRNA molecule in freckle whitening cosmetics or the preparation of medicines for treatment of diseases related to melanin gene. | 11-13-2014 |
20140350232 | SIRNA CONJUGATE AND PREPARATION METHOD THEREOF - Provided are an siRNA-polymer conjugate, and a method for preparing the same, and more specifically, to a hybrid conjugate formed by covalently bonding siRNA and a polymeric compound for improving the in vivo stability of siRNA, and to a preparation method of the hybrid conjugate. The conjugate of the present invention can improve the in vivo stability of siRNA, thereby achieving an efficient delivery of therapeutic siRNA into cells and exhibiting the activity of siRNA even with a small dose of a relative low concentration. Therefore, the conjugate can advantageously be used as not only an siRNA treatment tool for cancers and other infectious disease, but also a novel type siRNA delivery system. | 11-27-2014 |
20140350233 | SIRNA CONJUGATE AND PREPARATION METHOD THEREOF - Provided are an siRNA-polymer conjugate, and a method for preparing the same, and more specifically, to a hybrid conjugate formed by covalently bonding siRNA and a polymeric compound for improving the in vivo stability of siRNA, and to a preparation method of the hybrid conjugate. The conjugate of the present invention can improve the in vivo stability of siRNA, thereby achieving an efficient delivery of therapeutic siRNA into cells and exhibiting the activity of siRNA even with a small dose of a relative low concentration. Therefore, the conjugate can advantageously be used as not only an siRNA treatment tool for cancers and other infectious disease, but also a novel type siRNA delivery system. | 11-27-2014 |
20140357855 | MODULATION OF EXON RECOGNITION IN PRE-MRNA BY INTERFERING WITH THE SECONDARY RNA STRUCTURE - The invention relates to oligonucleotides for inducing skipping of exon 53 of the dystrophin gene. The invention also relates to methods of inducing exon 53 skipping using the oligonucleotides. | 12-04-2014 |
20140357856 | CHIMERIC OLIGOMERIC COMPOUNDS COMPRISING ALTERNATING REGIONS OF NORTHERN AND SOUTHERN CONFORMATIONAL GEOMETRY - The present invention relates to novel chimeric oligomeric compounds having a plurality of alternating regions having either RNA like having northern or 3′-endo conformational geometry (3′-endo regions) or DNA like having southern or C2′-endo/O4′-endo conformational geometry. The oligomeric compounds of the present invention have shown reduction in mRNA levels in multiple in vitro and in vivo assay systems and are useful, for example, for investigative and therapeutic purposes. | 12-04-2014 |
20140371439 | DOUBLE-STRANDED RNA COMPOUNDS TO CASP2 AND USES THEREOF - The present disclosure relates to methods of treating a patient suffering from or at risk of developing an ocular disease, disorder or injury, and includes treatment regimens using a double-stranded RNA compound that down-regulates CASP2 expression, or a pharmaceutically acceptable salt thereof. | 12-18-2014 |
20140371440 | SINGLE-STRANDED ANTIMICROBIAL OLIGONUCLEOTIDES AND USES THEREOF - The current invention is directed to oligonucleotide sequences isolated from a sequence designated rbl-1 [SEQ ID NO. 19] that either kill or inhibit growth, or prevent the production of endogenously expressed toxin, of microorganisms. These ssDNA sequences, identified through use of a screening method, appear to act as modulators of essential growth functions which may act at the level of triplex formation, antisense inhibition, or as aptamers that alter gene function. The sequences, referred to as minimum functional regions, or MFRs, are useful inter alia as therapeutic agents for treatment of sepsis and other pathologies caused by microorganisms such as sepsis and/or in which microorganisms are contributory agents. | 12-18-2014 |
20150011744 | Modifications for Antisense Compounds - The invention pertains to modifications for antisense oligonucleotides, wherein the modifications are used to improve stability and provide protection from nuclease degradation. The modifications could also be incorporated into double-stranded nucleic acids, such as synthetic siRNAs and miRNAs. | 01-08-2015 |
20150011745 | NUCLEIC ACID MOLECULE FOR INHIBITING ACTIVITY OF RNAI MOLECULE - The purpose of the present invention is to develop and provide a nucleic acid molecule that can specifically and efficiently inhibit the activity of a target RNAi molecule and can be produced safely at a low cost. Provided is a nucleic acid molecule for inhibiting the activity of a target RNAi molecule. The nucleic acid molecule comprises a single-stranded nucleic acid moiety that contains one unmodified DNA region composed of a nucleotide sequence completely or sufficiently complementary to a nucleotide sequence of a functional strand having the activity in the target RNAi molecule and a double-stranded nucleic acid moiety to be linked to at least one of the 5′-end and the 3′-end of the single-stranded nucleic acid moiety. | 01-08-2015 |
20150018539 | MODIFIED MIRNA AS A SCAFFOLD FOR SHRNA - What is described is a modified miRNA molecule for producing an artificial siRNA/mature small RNA molecule that inhibits the expression of a target transcript of a host cell, comprising a stem region modified to comprise a sequence encoding the artificial siRNA molecule, consisting of a guide and a passenger strand; a conserved region having specific sequences; and a nonconserved region modified to include a recognition site for a restriction enzyme while preserving the native secondary structure of the miRNA. The modified miRNA molecule produced with these elements substantially inhibits the expression of the target transcript when expressed from an endogenous or exogenous promoter in the host cell. | 01-15-2015 |
20150018540 | OLIGOMER-CONJUGATE COMPLEXES AND THEIR USE - Oligonucleotides, chemically-modified oligonucleotides, and oligonucleotide-conjugate compl in research, diagnostics, and/or therapeutics are described herein. | 01-15-2015 |
20150025232 | HEPTAMER-TYPE SMALL GUIDE NUCLEIC ACIDS INDUCING APOPTOSIS OF HUMAN LEUKEMIA CELLS - There is provided a heptamer-type small guide nucleic acid that comprises any of the 7-base sequences of SEQ ID NOS: 1 to 15, and induces apoptosis of human leukemia cells. A leukemia therapeutic agent containing the heptamer-type small guide nucleic acid as an active ingredient is also provided. The novel heptamer-type sg nucleic acid can induce apoptosis of human leukemia cells. | 01-22-2015 |
20150031871 | RNA-Mediated Gene Modulation - An isolated RNA comprising an intron RNA that is released in a cell, thereby modulating the function of a target gene. Also disclosed are a composition comprising a chemokine and an isolated RNA of the invention or a DNA template for the isolated RNA, a composition comprising one or more agents that induce RNA-mediated modulation of the functions of two or more target genes in a cell, and methods of using these compositions for modulating the functions of genes in a cell. | 01-29-2015 |
20150051389 | SELECTIVE ANTISENSE COMPOUNDS AND USES THEREOF - Disclosed are oligomeric compounds which are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. The hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell. | 02-19-2015 |
20150073133 | iRNA AGENTS WITH BIOCLEAVABLE TETHERS - The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose that further includes a tether having one or more linking groups, in which at least one of the linking groups is a cleavable linking group. The tether in turn can be connected to a selected moiety, e.g., a ligand, e.g., a targeting or delivery moiety, or a moiety which alters a physical property. The cleavable linking group is one which is sufficiently stable outside the cell such that it allows targeting of a therapeutically beneficial amount of an iRNA agent (e.g., a single stranded or double stranded iRNA agent), coupled by way of the cleavable linking group to a targeting agent—to targets cells, but which upon entry into a target cell is cleaved to release the iRNA agent from the targeting agent. | 03-12-2015 |
20150080563 | MODULATION OF EXON RECOGNITION IN PRE-MRNA BY INTERFERING WITH THE SECONDARY RNA STRUCTURE - The invention relates to oligonucleotides for inducing skipping of exon 55 of the dystrophin gene. The invention also relates to methods of inducing exon 55 skipping using the oligonucleotides. | 03-19-2015 |
20150080564 | SYNTHESIS AND USES OF NUCLEIC ACID COMPOUNDS WITH CONFORMATIONALLY RESTRICTED MONOMERS - Synthesis and uses of conformationally restricted nucleomonomers (CRN) to prepare nucleic acid compounds. Methods for preparing nucleomonomers for nucleic acid compounds in high yields and in multi-gram scale for therapeutic modalities useful for treating or preventing diseases or disorders by up- or down-regulating the expression of genes and other nucleic acid based regulatory systems in a cell. | 03-19-2015 |
20150094461 | OLIGOMERIC COMPOUNDS AND COMPOSITIONS FOR USE IN MODULATION OF SMALL NON-CODING RNAS - Compounds, compositions and methods are provided for modulating the expression and function of small non-coding RNAs. The compositions comprise oligomeric compounds, targeted to small non-coding RNAs. Methods of using these compounds for modulation of small non-coding RNAs as well as downstream targets of these RNAs and for diagnosis and treatment of disease associated with small non-coding RNAs are also provided. | 04-02-2015 |
20150105544 | POLYNUCLEOTIDE COMPRISING SEQUENCES OF WHEAT GLIADINS AND USE THEREOF FOR SILENCING BY RNAI - The present invention relates to the specific silencing of the α (alpha), β (beta), γ (gamma) and ω (omega)-gliadins of hard wheat for flour by RNA interference (RNAi) through employment of a polynucleotide which is transcribed into an hpRNA (hairpin RNA). Furthermore the present invention additionally relates to a vector, cell, plant or seed comprising the polynucleotide, the expression whereof is specifically directed in particular tissues of wheat seeds through gene expression-regulating sequences such as, for example, the promoter of a gene of γ-gliadins or the promoter of the gene encoding for a D-hordein. | 04-16-2015 |
20150105545 | INTERFERING RNA MOLECULES - The present invention is related to a ribonucleic acid comprising a double stranded structure whereby the double-stranded structure comprises a first strand and a second strand, whereby the first strand comprises a first stretch of contiguous nucleotides and whereby said first stretch is at least partially complementary to a target nucleic acid, and the second strand comprises a second stretch of contiguous nucleotides whereby said second stretch is at least partially identical to a target nucleic acid, and whereby the double stranded structure is blunt ended. | 04-16-2015 |
20150141636 | OLIGOMERIC COMPOUNDS COMPRISING BICYCLIC NUCLEOSIDES AND HAVING REDUCED TOXICITY - In certain embodiments, the present invention provides oligomeric compounds having favorable toxicity profiles and therapeutic indexes. Compounds of the present invention comprise bicyclic nucleosides. Certain such bicyclic nucleosides are pyrimidines that do not include a methyl group at the 5-carbon. Oligomeric compounds comprising such nucleosides are less toxic than compounds comprising bicyclic nucleosides that do include a methyl group at the 5-carbon. In certain embodiments, the present invention provides methods of preparing and using such compounds. | 05-21-2015 |
20150148529 | NUCLEIC ACID COMPLEX AND NUCLEIC ACID-POLYSACCHARIDE COMPLEX - As a means for solving the problem of providing a nucleic acid conjugate that does not undergo degradation at a DNA-RNA bonding site even in vivo, provided is a nucleic acid conjugate comprising a single-stranded DNA and a double-stranded RNA, wherein the 3′ position of the 3′-terminal deoxyribonucleotide residue of the single-stranded DNA is bonded to the 5′ position of the 5′-terminal ribonucleotide residue of one of the ribonucleotide strands of the double-stranded RNA, and the hydroxyl group at the 2′ position of the 5′-terminal nucleotide of the ribonucleotide strand, which is bonded to the single-stranded DNA, is substituted with an alkoxy group or a halogen atom, and/or the phosphate diester group between the 3′ position of the first ribonucleotide bonded to the single-stranded DNA and the 5′ position of the adjacent ribonucleotide is substituted with any of phosphorothioate group, dithiophosphate diester group and trithiophosphate diester group. | 05-28-2015 |
20150148530 | RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA) - The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism. | 05-28-2015 |
20150315575 | COMPOSITIONS AND METHODS FOR SELECTIVE DELIVERY OF OLIGONUCLEOTIDE MOLECULES TO CELL TYPES - The invention provides a conjugate comprising (i) a nucleic acid which is complementary to a target nucleic acid sequence and which expression prevents or reduces expression of the target nucleic acid and (ii) a selectivity agent which is capable of binding with high affinity to a receptor which can be internalised by the cell in response to the binding of said selectivity agent. The conjugates of the present invention are useful for the delivery of the nucleic acid to cell of interests and thus, for the treatment of diseases which require a down-regulation of the protein encoded by the target nucleic acid as well as for the delivery of contrast agents to the cells for diagnostic purposes. | 11-05-2015 |
20150315579 | DOUBLE STRAND COMPOSITIONS COMPRISING DIFFERENTIALLY MODIFIED STRANDS FOR USE IN GENE MODULATION - The present invention provides double stranded compositions wherein the first strand is modified to have a particular motif and the second strand is modified a selected motif. The motifs are defined by positioning of differentially modified nucleosides wherein at least the sugar moieties are different. More particularly, the present compositions comprise an antisense strand that is modified to have a positional/full motif and the sense strand is modified to have an alternating motif, a hemimer motif, a blockmer motif, a gapped motif, a positional motif, a positional/full motif or a fully modified motif. Each strand further comprises one or more phosphorothioate internucleoside linkage. The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes. In preferred embodiments the compositions of the present invention hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The present invention also provides methods for modulating gene expression. | 11-05-2015 |
20150315585 | MULTIMERIC OLIGONUCLEOTIDE COMPOUNDS HAVING NON-NUCLEOTIDE BASED CLEAVABLE LINKERS - The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties. Methods of making and uses of the described compounds are also provided | 11-05-2015 |
20150315587 | MULTIMERIC OLIGONUCLEOTIDES COMPOUNDS HAVING CLEAVABLE LINKERS - The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties. Methods of making and uses of the described compounds are also provided | 11-05-2015 |
20150315590 | ANTISENSE OLIGONUCLEOTIDES THAT TARGET A CRYPTIC SPLICE SITE IN USH1C AS A THERAPEUTIC FOR USHER SYNDROME - The present invention provides a method for treating Usher's syndrome in a human subject including administering to the human subject an oligonucleotide having 8 to 30 linked nucleosides having a nucleobase sequence comprising a complementary region comprising at least 8 contiguous nucleobases complementary to a target region of equal length within exon 3 of an Usher RNA transcript. | 11-05-2015 |
20150315594 | COMPOSITIONS AND METHODS FOR MODULATING ANGIOPOIETIN-LIKE 3 EXPRESSION - Provided herein are methods, compounds, and compositions for reducing expression of an ANGPTL3 mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for reducing lipids and/or glucose in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate any one or more of cardiovascular disease and/or metabolic disease, or a symptom thereof, in an individual in need thereof. | 11-05-2015 |
20150337313 | MUCOSAL HEALING PROMOTER - From experiments using colitis model mice, the present inventors discovered that siRNAs that suppress the CHST15 gene expression have a therapeutic effect against Crohn's disease or ulcerative colitis. Specifically, the present inventors discovered that the siRNAs which suppress the CHST15 gene expression can serve as an agent for promoting mucosal healing, in particular, an agent for treating Crohn's disease or ulcerative colitis, and thereby completed the present invention. | 11-26-2015 |
20150344879 | COMPOUNDS AND METHODS FOR MODULATING GENE EXPRESSION - The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing. | 12-03-2015 |
20150344886 | RNAi-Related Inhibition of TNF-alpha Signaling Pathway for Treatment of Ocular Angiogenesis - RNA interference is provided for inhibition of tumor necrosis factor α (TNFα) by silencing TNFα cell surface receptor TNF receptor-1 (TNFR1) mRNA expression, or by silencing TNFα converting enzyme (TACE/ADAM17) mRNA expression. Silencing such TNFα targets, in particular, is useful for treating patients having a TNFα-related condition or at risk of developing a TNFα-related condition, such as ocular angiogenesis, retinal ischemia, and diabetic retinopathy. | 12-03-2015 |
20150344887 | siRNA FOR INHIBITION OF USP15 EXPRESSION AND PHARMACEUTICAL COMPOSITION CONTAINING THE SAME - Disclosed herein is an siRNA that inhibits the expression of USP 15 protein and a pharmaceutical composition containing the same for preventing or treating cancers. According to the present invention, the siRNA of the present invention inhibits the expression of USP 15 protein thereby substantially inhibiting the growth and metastasis of cancer cells. | 12-03-2015 |
20150344892 | OLIGONUCLEOTIDES FOR MODULATION OF TARGET RNA ACTIVITY - The present invention relates to oligonucleotides for modulation of target RNA activity. Thus, the invention provides oligonucleotides that bind to microRNA binding sites of target RNA. The oligonucleotides may activate RNase H or RNAi. In a preferred embodiment, the oligonucleotides prevents a micro RNA from binding to its binding site of the target RNA and thereby prevent the microRNA from regulating the target RNA. Such oligonucleotides have uses in research and development of new therapeutics. | 12-03-2015 |
20150353929 | COMPOSITIONS AND METHODS FOR MODULATION OF SMN2 SPLICING - Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy. | 12-10-2015 |
20150353931 | ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHODS OF USE THEREOF - An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 214. | 12-10-2015 |
20150374842 | PROCESS FOR FORMULATING AN ANIONIC AGENT - Formulations comprising anionic agents such as nucleic acids within a lipid-containing particle methods of formulating a lipid-containing particle comprising an anionic agent such as a nucleic acid, methods for preparing a lipid-containing particle comprising an anionic agent such as a nucleic acid, methods for therapeutic delivery of an anionic agent to a patient in need thereof, where the anionic agent is formulated in a lipid-containing particle as described herein. | 12-31-2015 |
20150376611 | OLIGONUCLEOTIDE - The present invention provides an oligonucleotide having improved affinity for AGO2, and the like. The oligonucleotide has a nucleotide residue or a nucleoside residue represented by formula (I) {wherein X | 12-31-2015 |
20160002636 | ENA NUCLEIC ACID PHARMACEUTICALS CAPABLE OF MODIFYING SPLICING OF mRNA PRECURSORS - Oligonucleotides having a nucleotide sequence complementary to nucleotide numbers such as 2571-2607, 2578-2592, 2571-2592, 2573-2592, 2578-2596, 2578-2601 or 2575-2592 of the dystrophin cDNA (Gene Bank accession No. NM_004006.1) and therapeutic agents for muscular dystrophy comprising such oligonucleotides. | 01-07-2016 |
20160017323 | CONJUGATED ANTISENSE COMPOUNDS AND THEIR USE - Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the oligomeric compounds are conjugated to N-Acetylgalactosamine. | 01-21-2016 |
20160032293 | SELECTION OF RNA-APTAMERS AS ANTI-MALARIA AGENTS - The present invention relates to an aptamer or an active fragment thereof raised against the semi-conserved duffy binding ligand domain 1α, DBL1α, region of the | 02-04-2016 |
20160046937 | SMALL INTERFERENCE RNA FOR INHIBITING INTRACELLULAR EXPRESSION OF RIBOSOMAL PROTEIN - The present invention relates to a small interference RNA (siRNA) for specifically inhibiting the expression of ribosomal protein in cells, and more particularly, to an siRNA that specifically inhibits the expression of endogenous rpS3 protein in cells without influencing tagged overexpressed protein. The siRNAs of the present invention are excellent in terms of economy and efficiency compared to conventional commercially available products. | 02-18-2016 |
20160053263 | METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF BETA-CATENIN BY DOUBLE-STRANDED RNA - This invention relates to compounds, compositions, and methods useful for reducing β-catenin target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents. | 02-25-2016 |
20160053268 | ANTIPROLIFERATIVE AGENT - The invention provides an antibody specific to the ANGPTL4 protein capable of neutralizing proliferation and methods of making and using the same. The antibody of the invention is further directed to the C terminal region of the protein and may be capable of neutralizing cell proliferation and treating cancer. The antibody may be monoclonal and/or humanized antibody. | 02-25-2016 |
20160053269 | RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA) - The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism. | 02-25-2016 |
20160076040 | THERAPEUTIC COMPOSITIONS - This application relates to therapeutic siRNA agents and methods of making and using the agents. | 03-17-2016 |
20160102303 | METHOD OF ISOLATING NUCLEIC ACID - Disclosed herein is a method for isolating a nucleic acid from a sample. The method includes contacting the sample with boron carbide under conditions sufficient to form a boron carbide-nucleic acid complex. The complex is separated from the sample. | 04-14-2016 |
20160102310 | miR-96-5p INHIBITOR AND A SCREENING METHOD FOR THE INHIBITOR - The present invention provides novel medical means to facilitate glutathione (GSH) synthesis in the brain. These means are miR-96-5p inhibitor increasing GSH expression in the brain and a pharmaceutical composition comprising the miR-96-5p inhibitor and having a preventive and/or therapeutic performance to a disease caused by decrease of GSH amount or depression of GSH activity. | 04-14-2016 |
20160122372 | TRICYCLIC NUCLEOSIDES AND OLIGOMERIC COMPOUNDS PREPARED THEREFROM - The present invention provides novel tricyclic nucleosides and oligomeric compounds prepared therefrom. Incorporation of one or more of the tricyclic nucleosides into an oligomeric compound is expected to enhance one or more properties of the oligomeric compound. Such oligomeric compounds can also be included in double stranded compositions. In certain embodiments, the oligomeric compounds provided herein are expected to hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. | 05-05-2016 |
20160130587 | INTERFERING RNA MOLECULES - The present invention is related to a ribonucleic acid comprising a double stranded structure whereby the double-stranded structure comprises a first strand and a second strand, whereby the first strand comprises a first stretch of contiguous nucleotides and whereby said first stretch is at least partially complementary to a target nucleic acid, and the second strand comprises a second stretch of contiguous nucleotides whereby said second stretch is at least partially identical to a target nucleic acid, and whereby the double stranded structure is blunt ended. | 05-12-2016 |
20160138014 | ANTISENSE COMPOUNDS AND USES THEREOF - The present disclosure provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell. | 05-19-2016 |
20160138024 | Screening Method For Compound Having Obesity Preventive Or Therapeutic Activity | 05-19-2016 |
20160145620 | OLIGOMERS WITH IMPROVED OFF-TARGET PROFILE - The present invention provides oligomers that binds RNA that consists of a lower affinity region and a higher affinity region, wherein the monomers in the higher affinity region increases the melting temperature (i.e. affinity) of the oligomer base paired to RNA more than the monomers used in the lower affinity region, wherein said increase in melting temperature is relatively to the alternative use of DNA monomers of the same (base) sequence. The oligomers of the invention are useful for binding to target RNA such as mRNA and non-coding RNA and have the advantage that they will be less prone to off-target binding via the lower affinity region than via the higher affinity region. | 05-26-2016 |
20160168567 | UNA SINGLE STRANDED OLIGOMERS FOR THERAPEUTICS | 06-16-2016 |
20160168570 | MODULATION OF EXON RECOGNITION IN PRE-MRNA BY INTERFERING WITH THE SECONDARY RNA STRUCTURE | 06-16-2016 |
20160168575 | ANTISENSE DESIGN | 06-16-2016 |
20160168576 | ANTISENSE DESIGN | 06-16-2016 |
20160201065 | Methods and Compositions for Selecting siRNA of Improved Functionality | 07-14-2016 |
20160376585 | OLIGONUCLEOTIDE-LIGAND CONJUGATES AND PROCESS FOR THEIR PREPARATION - The present invention relates to ligand conjugates of oligonucleotides (e.g., iRNA agents) and methods for their preparation. The ligands are derived primarily from monosaccharides These conjugates are useful for the in vivo delivery of oligonucleotides. | 12-29-2016 |
20160376591 | iRNA AGENTS WITH BIOCLEAVABLE TETHERS - The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose that further includes a tether having one or more linking groups, in which at least one of the linking groups is a cleavable linking group. The tether in turn can be connected to a selected moiety, e.g., a ligand, e.g., a targeting or delivery moiety, or a moiety which alters a physical property. The cleavable linking group is one which is sufficiently stable outside the cell such that it allows targeting of a therapeutically beneficial amount of an iRNA agent (e.g., a single stranded or double stranded iRNA agent), coupled by way of the cleavable linking group to a targeting agent—to targets cells, but which upon entry into a target cell is cleaved to release the iRNA agent from the targeting agent. | 12-29-2016 |
20180023084 | Disulfide-Containing Alkyne Linking Agents | 01-25-2018 |
20180023087 | Screening Method For Compound Having Obesity Preventive Or Therapeutic Activity | 01-25-2018 |