Patent application number | Description | Published |
20090023599 | USE OF ANTISENSE OLIGONUCLEOTIDE LIBRARIES FOR IDENTIFYING GENE FUNCTION - A method for identifying one or more genes involved in a phenotype of cells, tissues or organisms, comprising the steps of contacting cells, tissues or organisms which exhibit the phenotype with a library of antisense oligonucleotides and performing a primary phenotypic assay to determine which antisense oligonucleotides in the library attenuate the phenotype. These antisense oligonucleotides correspond to genes involved in the phenotype. The method may be used to identify genes involved in various disease states. | 01-22-2009 |
20090029931 | Topical administrations of antisense compounds to vla-4 for the treatment of respiratory conditions - A method for the treatment and/or prophylaxis of an animal having a respiratory disease or condition associated with airway hyperresponsiveness, eosinophilia, neutrophilia, leukocytes or overproduction of mucus and/or with the expression of integrin α4 comprising administering to the animal a composition comprising from. 0.001 to 1000 μg per kg body weight of the animal of an antisense compound targeted to a nucleic acid molecule encoding integrin α4. | 01-29-2009 |
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 |
20090156526 | COMPOSITIONS AND METHODS FOR MODULATION OF LMNA EXPRESSION - Disclosed herein are compounds, compositions and methods for modulating the expression of LMNA in a cell, tissue or animal. Also provided are methods of target validation. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Further provided are methods of identifying cis splicing regulatory elements of a selected mRNA using the disclosed compounds. | 06-18-2009 |
20090264502 | COMPOSITIONS AND THEIR USES DIRECTED TO HSP27 - Disclosed herein are compounds, compositions and methods for modulating the expression of HSP27 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. | 10-22-2009 |
20090286969 | 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. | 11-19-2009 |
20090291906 | 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. | 11-26-2009 |
20090291907 | 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. | 11-26-2009 |
20090298174 | 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. | 12-03-2009 |
20090317907 | 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. | 12-24-2009 |
20100041047 | EFFICIENT REDUCTION OF TARGET RNA'S BY SINGLE- AND DOUBLE-STRANDED OLIGOMERIC COMPOUNDS - The present invention provides, inter alia, methods of selecting a single-stranded oligomeric compounds for inhibiting RNA expression, methods of generating double-stranded oligomeric compounds, methods of identifying optimized double-stranded oligomeric compounds, methods of selecting optimized single-stranded oligomeric compounds, methods of selecting optimized double-stranded oligomeric compounds, methods of identifying multifunctional oligomeric compounds, methods for optimizing target region selection for modulation of RNA expression, methods of optimizing expression modulation of RNA, and the like. The present invention further provides oligomeric compounds, 8-80 nucleobases in length targeted to a target RNA, wherein said oligomeric compound hybridizes to said target RNA and inhibits RNA levels by at least 50% in both single-stranded and double-stranded forms, and multifunctional oligomeric compounds. | 02-18-2010 |
20100056607 | COMPOSITIONS AND THEIR USES DIRECTED TO HBXIP - Disclosed herein are compounds, compositions and methods for modulating the expression of HBXIP in a cell, tissue or animal. Also provided are methods of target validation. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. | 03-04-2010 |
20100069472 | 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 progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntingtin's disease in an individual susceptible to Huntingtin's Disease. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. | 03-18-2010 |
20100081705 | METHODS FOR SLOWING FAMILIAL ALS DISEASE PROGRESSION - Methods for slowing disease progression in an individual suffering from familial ALS are provided. Also provided are methods of increasing the survival time of an individual suffering from familial ALS. These methods employ antisense oligonucleotides targeted to SOD1, for use in inhibiting the expression of SOD1 in the central nervous system of an individual suffering from familial ALS. | 04-01-2010 |
20100130595 | ANTISENSE OLIGONUCLEOTIDES DIRECTED AGAINST CONNECTIVE TISSUE GROWTH FACTOR AND USES THEREOF - This invention provides compounds which comprise modified oligonucleotides capable of inhibitory expression of connective tissue factor and composition containing same as well as methods of treating hyperprolific disorders and fibrotic diseases, and of reducing scarring resulting from wound healing using such compounds. | 05-27-2010 |
20100152280 | MODULATION OF SID-1 EXPRESSION - Compounds, compositions and methods are provided for modulating the expression of SID-1. The compositions comprise oligonucleotides, targeted to nucleic acid encoding SID-1. Methods of using these compounds for modulation of SID-1 expression and for diagnosis and treatment of diseases and conditions associated with expression of SID-1 are provided. | 06-17-2010 |
20100222231 | USE OF ANTISENSE OLIGONUCLEOTIDE LIBRARIES FOR IDENTIFYING GENE FUNCTION - A method for identifying one or more genes involved in a phenotype of cells, tissues or organisms, comprising the steps of contacting cells, tissues or organisms which exhibit the phenotype with a library of antisense oligonucleotides and performing a primary phenotypic assay to determine which antisense oligonucleotides in the library attenuate the phenotype. These antisense oligonucleotides correspond to genes involved in the phenotype. The method may be used to identify genes involved in various disease states. | 09-02-2010 |
20100267813 | 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. | 10-21-2010 |
20100267814 | 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. | 10-21-2010 |
20100292304 | COMPOUNDS AND METHODS FOR IMPROVING CELLULAR UPTAKE OF OLIGOMERIC COMPOUNDS - The present invention provides method of optimizing the efficacy and potency of antisense drugs. In certain embodiments, the invention provides assays useful for determining favorable oligonucleotide characteristics and excipeints for improved cellular uptake. | 11-18-2010 |
20110039334 | CHIMERIC OLIGOMERIC COMPOUNDS FOR MODULATION OF SPLICING - Disclosed herein are compounds, compositions and methods for modulating splicing of a selected target mRNA. Further provided are uses of the disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Methods of enhancing cellular uptake, modulating tissue distribution and enhancing pharmacological activity of RNase H-independent antisense oligonucleotides are also provided. | 02-17-2011 |
20110054004 | METHOD FOR REDUCING SCARRING DURING WOUND HEALING USING ANTISENSE COMPOUNDS DIRECTED TO CTGF - This invention provides a method for reducing hypertropic scarring resulting from dermal wound healing in a subject in need which comprises administering to the subject an antisense oligonucleotide which inhibits expression of connective tissue growth factor (CTGF) in an amount effective to inhibit expression of CTGF and thereby reduce hypertrophic scarring. | 03-03-2011 |
20110124591 | 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. | 05-26-2011 |
20110124715 | Antiviral Oligonucleotides Having a Conserved G4 Core Sequence - Modified oligonucleotides having a conserved G | 05-26-2011 |
20110160283 | MODULATION OF CD40 EXPRESSION - Disclosed herein are antisense compounds and methods for decreasing CD40. Examples of disease conditions that can be ameliorated with the administration of antisense compounds targeted to CD40 include hyperproliferative disorders, graft versus host disease (GVHD), graft rejection, asthma, airway hyperresponsiveness, chronic obstructive pulmonary disease (COPD), multiple sclerosis (MS), systemic lupus erythematosus (SLE), and certain forms of arthritis. | 06-30-2011 |
20110171730 | Conjugates For Use In Hepatocyte Free Uptake Assays - The present invention provides methods of identifying oligomeric compounds, such as siRNA and double-stranded RNA compounds, having bioactivity in vivo, and kits. | 07-14-2011 |
20110172296 | MODULATION OF TRANSFORMING GROWTH FACTOR-BETA 1 EXPRESSION - Provided are compounds capable of inhibiting expression of TGF-beta 1 and compositions containing same as well as methods using such compounds for treating fibrotic diseases including the reduction of scarring resulting from wound healing. | 07-14-2011 |
20110213011 | MODULATION OF SMAD3 EXPRESSION - Provided are compounds capable of inhibiting SMAD3 and compositions containing same as well as methods using such compounds for treating fibrosis and scarring. | 09-01-2011 |
20110224277 | 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. | 09-15-2011 |
20110269818 | MODULATION OF PRION EXPRESSION - Disclosed herein are compounds and methods for decreasing PrP and preventing, ameliorating, or treating a prion disease or conformational neurodegenerative disorder, in an individual in need thereof. Examples of disease conditions that can be ameliorated with the administration of antisense compounds targeted to PrP include Creutzfeldt-Jakob disease (CJD); variant Creutzfeldt-Jakob Disease (vCJD); Gerstmann-Straussler-Scheinker syndrome; fatal familial insomnia; kuru; Bovine Spongiform Encephalopathy (BSE), e.g. “mad cow disease”; Chronic Wasting Disease (CWD); scrapie; transmissible mink encephalopathy; feline spongiform encephalopathy; ungulate spongiform encephalopathy; Alzheimer's disease; Parkinson's disease; Huntington's disease; and Amyotrophic Lateral Sclerosis (ALS). | 11-03-2011 |
20120029049 | Antisense Modulation of Superoxide Dismutase 1, Soluble Expression - Antisense compounds, compositions and methods are provided for modulating the expression of superoxide dismutase 1, soluble. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding superoxide dismutase 1, soluble. Methods of using these compounds for modulation of superoxide dismutase 1, soluble expression and for treatment of diseases associated with expression of superoxide dismutase 1, soluble are provided. | 02-02-2012 |
20120035248 | METHOD FOR INHIBITING THE ACTIVITY OF MIR-155 - 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. | 02-09-2012 |
20120115930 | COMPOSITIONS AND THEIR USES DIRECTED TO HEPCIDIN - Disclosed herein are compounds, compositions and methods for modulating the expression of hepcidin in a cell, tissue or animal or preventing, ameliorating or treating anemia. Also provided are methods for prevention, amelioration or treatment of anemia, and for increasing red blood cell count in an animal. Also provided are methods for the prevention, amelioration and/or treatment of low serum iron levels, low red blood cell count and other clinical endpoints of anemia in an animal. These methods may be achieved by administration of compounds or compositions including antisense compounds targeted to a nucleic acid that expresses hepcidin polypeptide combined with an erythropoiesis stimulating agent. | 05-10-2012 |
20120122216 | 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. | 05-17-2012 |
20120149757 | 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. | 06-14-2012 |
20120157514 | 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. | 06-21-2012 |
20120184031 | ANTISENSE INHIBITION VIA RNASE H-INDEPENDENT REDUCTION IN MRNA - The present invention provides compositions and methods for reducing levels of a preselected mRNA, using antisense compounds targeted to a splice site or a region up to 50 nucleobases upstream of an exon/intron junction on said mRNA. Preferably, said antisense compounds do not elicit RNAse H cleavage of the mRNA. | 07-19-2012 |
20120190728 | COMPOSITIONS AND METHODS FOR MODULATION OF SMN2 SPLICING IN A SUBJECT - Disclosed herein are compounds, compositions and methods for modulating splicing of SMN | 07-26-2012 |
20120214865 | METHODS FOR SLOWING FAMILIAL ALS DISEASE PROGRESSION - Methods for slowing disease progression in an individual suffering from familial ALS are provided. Also provided are methods of increasing the survival time of an individual suffering from familial ALS. These methods employ antisense oligonucleotides targeted to SOD1, for use in inhibiting the expression of SOD1 in the central nervous system of an individual suffering from familial ALS. | 08-23-2012 |
20120252879 | MODULATION OF HUNTINGTIN EXPRESSION - Provided herein are methods, compounds, and compositions for reducing expression of huntingtin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate Huntington's disease, or a symptom thereof. | 10-04-2012 |
20120264806 | OLIGOMERIC COMPOUNDS AND EXCIPIENTS - The present invention provides method of optimizing the efficacy and potency of antisense compounds. In certain embodiments, the invention provides assays useful for determining favorable oligonucleotide characteristics and excipients for improved cellular uptake. | 10-18-2012 |
20120283319 | 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. | 11-08-2012 |
20120295962 | 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. | 11-22-2012 |
20130035370 | COMPOSITIONS AND METHODS FOR MODULATION OF LMNA EXPRESSION - Disclosed herein are compounds, compositions and methods for modulating the expression of LMNA in a cell, tissue or animal. Also provided are methods of target validation. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Further provided are methods of identifying cis splicing regulatory elements of a selected mRNA using the disclosed compounds. | 02-07-2013 |
20130046007 | SELECTIVE REDUCTION OF ALLELIC VARIANTS - Disclosed herein are antisense compounds and methods for selectively of reducing expression of an allelic variant of a gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate diseases, including neurodegenerative, such as Huntington's Disease (HD). | 02-21-2013 |
20130046008 | SELECTIVE REDUCTION OF ALLELIC VARIANTS - Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a huntingtin gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate Huntington's Disease (HD). | 02-21-2013 |
20130059902 | METHODS AND COMPOSITIONS USEFUL IN TREATMENT OF DISEASES OR CONDITIONS RELATED TO REPEAT EXPANSION - The present invention is drawn to chemically-modified oligomers that are complementary to, and capable of hybridizing within the repeat region of CAG, CUG, or CCUG nucleotide repeat-containing RNAs (NRRs). | 03-07-2013 |
20130164844 | COMPOUNDS AND METHODS FOR IMPROVING CELLULAR UPTAKE OF OLIGOMERIC COMPOUNDS - The present invention provides method of optimizing the efficacy and potency of antisense drugs. In certain embodiments, the invention provides assays useful for determining favorable oligonucleotide characteristics and excipeints for improved cellular uptake. | 06-27-2013 |
20130189782 | 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 progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntingtin's disease in an individual susceptible to Huntingtin's Disease. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. | 07-25-2013 |
20130190380 | ANTISENSE OLIGONUCLEOTIDES DIRECTED AGAINST CONNECTIVE TISSUE GROWTH FACTOR AND USES THEREOF - This invention provides compounds which comprise modified oligonucleotides capable of inhibitory expression of connective tissue factor and composition containing same as well as methods of treating hyperprolific disorders and fibrotic diseases, and of reducing scarring resulting from wound healing using such compounds. | 07-25-2013 |
20130190382 | METHODS FOR INHIBITING EXPRESSION OF CONNECTIVE TISSUE GROWTH FACTOR - This invention provides compounds which comprise modified oligonucleotides capable of inhibitory expression of connective tissue factor and composition containing same as well as methods of treating hyperprolific disorders and fibrotic diseases, and of reducing scarring resulting from wound healing using such compounds. | 07-25-2013 |
20130225659 | MODULATION OF NUCLEAR-RETAINED RNA - Provided herein are methods, compounds, and compositions for reducing expression of a nrRNA in an animal. Also provided herein are methods, compounds, and compositions for treating, ameliorating, delaying or reducing a symptom of a disease or disorder associated with a nuclear-retained RNA in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate a disease or condition associated with a nuclear-retained RNA, or a symptom thereof. | 08-29-2013 |
20130237585 | MODULATION OF DYSTROPHIA MYOTONICA-PROTEIN KINASE (DMPK) EXPRESSION - Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof. | 09-12-2013 |
20130289092 | COMPOUNDS AND METHODS FOR MODULATING INTERACTION BETWEEN PROTEINS AND TARGET NUCLEIC ACIDS - Provided herein are antisense compounds and methods for recruiting one or more non-cleaving protein to a target nucleic acid in a cell. In certain instances such recruitment of a non-cleaving protein alters the function or activity of the target nucleic acid. In certain such instances, the target nucleic acid a pre-mRNA and the recruitment of the non-cleaving protein results in a change in splicing of the pre-mRNA. | 10-31-2013 |
20140005252 | MODULATION OF ALPHA SYNUCLEIN EXPRESSION | 01-02-2014 |
20140005374 | CHIMERIC OLIGOMERIC COMPOUNDS FOR MODULATION OF SPLICING | 01-02-2014 |
20140057963 | 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. | 02-27-2014 |
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 |
20140121364 | 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 |
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 |
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 |
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 |
20140309282 | COMPOSITIONS AND METHODS FOR MODULATION OF LMNA EXPRESSION - Disclosed herein are compounds, compositions and methods for modulating the expression of LMNA in a cell, tissue or animal. Also provided are methods of target validation. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Further provided are methods of identifying cis splicing regulatory elements of a selected mRNA using the disclosed compounds. | 10-16-2014 |
20140329882 | 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. | 11-06-2014 |
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 |
20140357558 | COMPOSITIONS AND METHODS FOR TREATMENT OF SPINAL MUSCULAR ATROPHY - Disclosed herein are compounds, compositions and methods for treatment of diseases and disorders, including spinal muscular atrophy. | 12-04-2014 |
20140371296 | METHODS FOR MODULATING METASTASIS-ASSOCIATED-IN-LUNG-ADENOCARCINOMA-TRANSCRIPT-1 (MALAT-1) EXPRESSION - The present embodiments provide compounds and methods for reducing expression of Metastasis-Associated-in-Lung-Adenocarcinoma-Transcript-1 (MALAT-1) RNA and/or protein in an animal. Such methods are useful for treating cancer, such as colon cancer, intestinal cancer, lung cancer (e.g. non-small cell lung cancer), liver cancer, and/or prostate cancer. In various aspects, the cancer is a primary cancer. | 12-18-2014 |
20150025231 | COMPOSITIONS AND METHODS FOR MODULATION OF IKBKAP SPLICING - The present disclosure provides compounds comprising oligonucleotides complementary to a portion of the IKBKAP gene. Certain such compounds are useful for hybridizing to a portion of the IKBKAP gene, including but not limited to a portion of the IKBKAP gene in a cell. In certain embodiments, such hybridization results in modulation of splicing of the IKBKAP gene. In certain embodiments, the IKBKAP gene includes a mutation that results in defective splicing and a truncated IKAP protein. In certain embodiments, hybridization of oligonucleotides complementary to a portion of the IKBKAP gene results in a decrease in the amount of defective splicing and truncated IKAP protein. In certain embodiments, hybridization of oligonucleotides complementary to a portion of the IKBKAP gene results in an increase in the amount of normal splicing and functional, full-length IKAP protein. In certain embodiments, oligonucleotides are used to treat Familial Dysautonomia. | 01-22-2015 |