THE BROAD INSTITUTE, INC. Patent applications |
Patent application number | Title | Published |
20160125128 | ACCURATE TYPING OF HLA THROUGH EXOME SEQUENCING - Embodiments of the present disclosure present bioinformatic systems, methods, for allelic HLA typing (as well as and computer readable media having instructions for performing methods of HLA typing) using, for example, Illumina exome-sequencing data (e.g., 101 basepair, paired-end reads). | 05-05-2016 |
20160116474 | COMPOSITIONS AND METHODS FOR DETECTING AND TREATING GLIOBLASTOMA - The present invention provides compositions and methods for the diagnosis and treatment of glioblastoma, particularly tumor propagating cells within the glioblastoma. | 04-28-2016 |
20160060699 | SLE AND SLE-RELATED DISEASE-ASSOCIATED RISK MARKERS AND USES THEREOF - Provided herein are methods and compositions for identifying subjects, including canine subjects, as having an elevated risk of developing systemic lupus erythematosus (SLE) or an SLE-related immune-mediated rheumatic disorder or having undiagnosed SLE or an SLE-related immune-mediated rheumatic disorder. These subjects are identified based on the presence of gem-line risk markers. | 03-03-2016 |
20160032397 | MAST CELL CANCER-ASSOCIATED GERM-LINE RISK MARKERS AND USES THEREOF - Provided herein are methods and compositions for identifying subjects, including canine subjects, as having an elevated risk of developing cancer or having an undiagnosed cancer. These subjects are identified based on the presence of germ-line risk markers. | 02-04-2016 |
20160031837 | COMPOUNDS FOR INDUCING PROLIFERATION AND DIFFERENTIATION OF CELLS, AND METHODS OF USE THEREOF - The present invention provides methods of inducing proliferation of and/or differentiating cells comprising contacting cells with compounds within the methods of the invention. The present invention further provides cells obtainable by the methods of the invention. Liver disease affects more than 500 million people worldwide. Organ transplantation is the gold standard for treatment of liver failure, but organ shortages are acute. | 02-04-2016 |
20160024589 | METHODS OF IDENTIFYING RESPONSES TO MAP KINASE INHIBITION THERAPY - The invention provides methods and devices for determining molecular signatures in a cancer that predict response to a MARPK pathway inhibitor and methods of use of such signatures. | 01-28-2016 |
20160024588 | OSTEOSARCOMA-ASSOCIATED RISK MARKERS AND USES THEREOF - Provided herein are methods and compositions for identifying subjects, including canine subjects, as having an elevated risk of developing cancer or having an undiagnosed osteosarcoma. These subjects are identified based on the presence of germ-line risk markers. | 01-28-2016 |
20160016913 | COMPOUNDS AND COMPOSITIONS FOR THE TREATMENT OF CANCER - Disclosed are compounds, such as pyridazinones, that can be, inter alia, used for treating cancer. | 01-21-2016 |
20150376702 | METHODS FOR ASSESSING THE RISK OF CANINE ATOPIC DERMATITIS - The present invention relates to methods for assessing the risk of a dog to develop canine atopic dermatitis. The methods comprise detecting in a sample of DNA obtained from a dog the presence or absence of at least one genetic marker, wherein said at least one genetic marker is located on dog ( | 12-31-2015 |
20150353503 | INDAZOLE DERIVATIVES AND USES THEREOF - The present invention provides novel compounds (e.g., compounds of Formula (I)), and pharmaceutically acceptable salts, solvates, hydrates, polymorphs, co-crystals, tautomers, stereoisomers, isotopically labeled derivatives, prodrugs, and compositions thereof. Also provided are methods and kits comprising the inventive compounds, or compositions thereof, for treating and/or preventing a fungal or protozoan infection, inhibiting the activity of a fungal or protozoan enzyme, killing a fungus or protozoon, or inhibiting the growth of a fungus or protozoon. The fungus may be a | 12-10-2015 |
20150338425 | TREATMENT AND PROGNOSIS OF LYMPHANGIOLEIOMYOMATOSIS - Embodiments disclosed herein relate to treatment methods, diagnosis methods, drug efficacy evaluation methods and prognosis evaluation methods of lymphangioleiomyomatosis (LAM) and a disease associated with a mutation in a tuberous sclerosis complex (TSC) gene. The methods comprising analyses of the levels of lysophosphatidylcholine (LPC) in a biological sample of a subject, or comprising analyses of images of the location and signal intensity of isotope-labeled choline in the subject. | 11-26-2015 |
20150313890 | KINASE INHIBITORS AND METHODS OF USE THEREOF - The present invention provides compounds of formula I, pharmaceutically acceptable salts thereof, and pharmaceutical compositions thereof. Compounds of the present invention are useful for inhibiting kinase (e.g., GSK3 (e.g., GSK3α or GSK3β) or CK1) activity. The present invention further provides methods of using the compounds described herein for treating kinase-mediated disorders, such as neurological diseases, psychiatric disorders, metabolic disorders, and cancer. | 11-05-2015 |
20150299795 | CANCER-ASSOCIATED GERM-LINE AND SOMATIC MARKERS AND USES THEREOF - The invention provides methods and compositions for identifying subjects, including canine subjects, having an elevated risk of developing cancer or having an undiagnosed cancer. These subjects are identified based on the presence of germ-line allele(s) and markers and various somatic mutations. | 10-22-2015 |
20150259298 | THIOHYDANTOIN DERIVATIVES AND USES THEREOF - The present invention provides novel compounds (e.g., compounds of Formula (I)), and pharmaceutically acceptable salts, solvates, hydrate, polymorphs, co-crystals, tautomers, stereoisomers, isotopically labeled derivatives, prodrugs, and compositions thereof. Also provided are methods and kits comprising the inventive compounds, or compositions thereof, for treating and/or preventing a fungal or protozoan infection, inhibiting the activity of a fungal or protozoan enzyme, killing a fungus or protozoan, or inhibiting the growth of a fungus or protozoan. The fungus may be a | 09-17-2015 |
20150197785 | METHODS AND APPARATUS FOR ANALYZING AND QUANTIFYING DNA ALTERATIONS IN CANCER - Methods and apparatus for inferring purity and ploidy from a sample of cells (e.g., a sample comprising cancer and normal cells) are described. Copy number per cell of interest (e.g., cancer cell) is determined by optimizing purity and ploidy for the sample based, at least in part, on relative copy number profile information. One or more likelihood fit scores are determined for each of a plurality of candidate solutions generated by the methods described herein. A solution is selected based, at least in part on the likelihood fit score(s) and the copy number per cancer cell is determined in accordance with the selected solution. | 07-16-2015 |
20150191427 | INHIBITORS OF HISTONE DEACETYLASE - The present invention relates to compounds of formula (I) or a pharmaceutically acceptable salt, hydrate, solvate, or prodrug thereof, wherein X | 07-09-2015 |
20150174138 | DIAGNOSTIC AND TREATMENT METHODS IN SUBJECTS HAVING OR AT RISK OF DEVELOPING RESISTANCE TO CANCER THERAPY - The invention relates to methods of treatment and diagnosis of subjects with cancer. In some aspects, the invention relates to methods of treatment and diagnosis of subjects with cancer, wherein the cancer is characterized by a Notch pathway activation mutation or by resistance to a Notch pathway inhibitor. | 06-25-2015 |
20150141470 | DIAGNOSTIC AND TREATMENT METHODS IN PATIENTS HAVING OR AT RISK OF DEVELOPING RESISTANCE TO CANCER THERAPY - A method of identifying a subject having cancer who is likely to benefit from treatment with a combination therapy with a MAPK pathway inhibitor, such as a RAF inhibitor, MEK inhibitor, or ERK inhibitor, and a GEF or HDAC inhibitor is provided. A method of treating cancer in a subject in need thereof is also provided and includes administering to the subject an effective amount of a MAPK inhibitor, such as a RAF inhibitor, MEK inhibitor, or ERK inhibitor, and an effective amount of a GEF or HDAC inhibitor. A method of identifying targets that confers resistance to a MAPK pathway inhibitor is also provided. | 05-21-2015 |
20150086565 | CANCER-RELATED EXTRACELLULAR MATRIX SIGNATURES AND RELATED METHODS AND PRODUCTS - Proteomic methods for identifying cancer related proteins and related products and kits are provided. The cancer specific proteins are extracellular matrix proteins that are associated with various aspects of cancer. Panels or signature sets of proteins useful in the detection, diagnosis and treatment of cancers as well as monitoring therapeutic progress in a cancer patient are provided herein along with methods for their detection and for their use in targeting imaging and/or therapeutic agents to the tumors via binding to the specified proteins. The proteins were identified using proteomics analyses of tissue samples taken from cancer patients. In certain aspects the proteins are particularly useful in colon cancer patients. | 03-26-2015 |
20150044676 | MULTIPLEX METHODS TO ASSAY MIXED CELL POPULATIONS SIMULTANEOUSLY - Methods to simultaneously test and screen multiplexed, mixed cell populations, e.g., populations comprising genetically heterogeneous cancer cells, in common conditions. | 02-12-2015 |
20140364439 | MARKERS ASSOCIATED WITH CHRONIC LYMPHOCYTIC LEUKEMIA PROGNOSIS AND PROGRESSION - The present invention provides methods and devices related to markers (or biomarkers) associated with chronic lymphocytic leukemia (CLL). Examples of these markers include drivers of CLL progression. The invention contemplates, inter alia, detecting the clonal, including subclonal, profile of CLL in a subject and the presence (or absence) of subclonal driver mutations, and utilizing this information in predicting disease progression, need, timing and/or nature of treatment regimen, and likelihood and frequency of relapse. | 12-11-2014 |
20140348819 | Methods of Treating Cancer - The present invention provides methods of treating cancer. | 11-27-2014 |
20140335620 | ENGINEERING AND OPTIMIZATION OF IMPROVED SYSTEMS, METHODS AND ENZYME COMPOSITIONS FOR SEQUENCE MANIPULATION - The invention provides for engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are compositions and methods related to components of a CRISPR complex particularly comprising a Cas ortholog enzyme. | 11-13-2014 |
20140335550 | FLUORESCENT SUBSTRATES FOR DETERMINING LYSINE MODIFYING ENZYME ACTIVITY - The invention relates to a compound of Formula I: | 11-13-2014 |
20140296232 | COMPOUNDS FOR THE TREATMENT OF MYCOBACTERIAL INFECTIONS - The invention relates to compounds of Formula I or a pharmaceutically acceptable salt, ester or prodrug thereof: | 10-02-2014 |
20140287938 | RECOMBINANT VIRUS AND PREPARATIONS THEREOF - The present invention generally relates to methods and compositions used delivery of gene editing compositions including transcriptional effectors with parvovirus and preferred methods for making same. | 09-25-2014 |
20140275268 | NOVEL SOLID FORMS OF TACEDINALINE - Novel solid forms of tacedinaline (4-(acetylamino)-N-(2-aminophenyl)benzamide), including crystalline tacedinaline Form B, a novel crystalline tacedinaline TFA salt, and amorphous tacedinaline, are disclosed. Pharmaceutical compositions comprising crystalline tacedinaline Form B, the novel crystalline tacedinaline TFA salt, and/or amorphous tacedinaline, and methods of treating various conditions by administering those novel solid forms, are also disclosed. | 09-18-2014 |
20140273232 | ENGINEERING OF SYSTEMS, METHODS AND OPTIMIZED GUIDE COMPOSITIONS FOR SEQUENCE MANIPULATION - The invention provides for systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for selecting specific cells by introducing precise mutations utilizing the CRISPR-Cas system. | 09-18-2014 |
20140273231 | CRISPR-CAS COMPONENT SYSTEMS, METHODS AND COMPOSITIONS FOR SEQUENCE MANIPULATION - The invention provides for systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for selecting specific cells by introducing precise mutations utilizing the CRISPR/Cas system. | 09-18-2014 |
20140271602 | NUCLEOTIDE-SPECIFIC RECOGNITION SEQUENCES FOR DESIGNER TAL EFFECTORS - The invention relates to methods of altering expression of a genomic locus of interest or specifically targeting a genomic locus of interest in an animal cell, which may involve contacting the genomic locus with a non-naturally occurring or engineered composition that includes a deoxyribonucleic acid (DNA) binding polypeptide having a N-terminal capping region, a DNA binding domain comprising at least five or more Transcription activator-like effector (TALE) monomers and at least one or more half-monomers specifically ordered to target the genomic locus of interest, and a C-terminal capping region, wherein the polypeptide includes at least one or more effector domains, and wherein the polypeptide is encoded by and translated from a codon optimized nucleic acid molecule so that the polypeptide preferentially binds to the DNA of the genomic locus. | 09-18-2014 |
20140256767 | DIRECT INHIBITORS OF KEAP1-NRF2 INTERACTION AS ANTIOXIDANT INFLAMMATION MODULATORS - A method of identifying compounds as direct inhibitors of Keap1-Nrf2 interaction through high-throughput screening and lead development. The direct inhibitors of Keap1-Nrf2 interaction are more specific and free of various undesirable effects than existing indirect inhibitors, and are potential drug candidates of chemopreventive and therapeutic agents for treatment of various diseases or conditions involving oxidative stress and/or inflammation, including but not limited to cancers, diabetes, Alzheimer's, and Parkinson's. Novel compounds are identified and methods of preventing or treating diseases or conditions related to Keap1-Nrf2 interaction activity by use of the novel compounds identified or compositions containing such compounds are also disclosed. | 09-11-2014 |
20140256046 | ENGINEERING AND OPTIMIZATION OF SYSTEMS, METHODS AND COMPOSITIONS FOR SEQUENCE MANIPULATION WITH FUNCTIONAL DOMAINS - The invention provides for engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors with additional functional domains. Also provided are methods of directing CRISPR complex formation in prokaryotic and eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity. | 09-11-2014 |
20140248702 | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes - The invention provides for systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for selecting specific cells by introducing precise mutations utilizing the CRISPR/Cas system. | 09-04-2014 |
20140243242 | COMPOSITIONS AND METHODS FOR CO-AMPLIFYING SUBSEQUENCES OF A NUCLEIC ACID FRAGMENT SEQUENCE - The present invention is related to genomic nucleotide sequencing. In particular, the invention describes a single reaction method to co-amplify multiple subsequences of a nucleic acid fragment sequence (i.e., for example, at least two read pairs from a single library insert sequence). Nucleic acid fragment sequences may include, but are not limited to, localizing library insert sequences and/or unique read pair sequences in specific orientations on a single emulsion polymerase chain reaction bead. Methods may include, but are not limited to, annealing, melting, digesting, and/or reannealing high throughput sequencing primers to high throughput sequencing primer binding sites. The compositions and methods disclosed herein contemplate sequencing complex genomes, amplified genomic regions, as well as detecting chromosomal structural rearrangements that are compatible with massively parallel high throughput sequencing platforms as well as ion semiconductor matching sequencing platforms (i.e., for example, Ion Torrent platforms). | 08-28-2014 |
20140242700 | ENGINEERING AND OPTIMIZATION OF IMPROVED SYSTEMS, METHODS AND ENZYME COMPOSITIONS FOR SEQUENCE MANIPULATION - The invention provides for engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are compositions and methods related to components of a CRISPR complex particularly comprising a Cas ortholog enzyme. | 08-28-2014 |
20140242699 | DELIVERY, ENGINEERING AND OPTIMIZATION OF SYSTEMS, METHODS AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND THERAPEUTIC APPLICATIONS - The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition. | 08-28-2014 |
20140242664 | ENGINEERING OF SYSTEMS, METHODS AND OPTIMIZED GUIDE COMPOSITIONS FOR SEQUENCE MANIPULATION - The invention provides for systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for selecting specific cells by introducing precise mutations utilizing the CRISPR-Cas system. | 08-28-2014 |
20140234972 | CRISPR-CAS Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes - The invention provides for systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for selecting specific cells by introducing precise mutations utilizing the CRISPR/Cas system. | 08-21-2014 |
20140200163 | MULTIPLEXED GENETIC REPORTER ASSAYS AND COMPOSITIONS - The invention provides methods for determining the activity of a plurality of nucleic acid regulatory elements. These methods may facilitate, e.g., the systematic reverse engineering, and optimization of mammalian cis-regulatory elements at high resolution and at a large scale. The method may include integration of multiplexed DNA synthesis and sequencing technologies to generate and quantify the transcriptional regulatory activity of e.g., thousands of arbitrary DNA sequences in parallel in cell-based as says (e.g., mammalian cell based assays). | 07-17-2014 |
20140186843 | METHODS, SYSTEMS, AND APPARATUS FOR IDENTIFYING TARGET SEQUENCES FOR CAS ENZYMES OR CRISPR-CAS SYSTEMS FOR TARGET SEQUENCES AND CONVEYING RESULTS THEREOF - Disclosed are locational or positional methods concerning CRISPR-Cas systems, and apparatus therefor. | 07-03-2014 |
20140179770 | DELIVERY, ENGINEERING AND OPTIMIZATION OF SYSTEMS, METHODS AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND THERAPEUTIC APPLICATIONS - The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition. | 06-26-2014 |
20140179006 | CRISPR-CAS COMPONENT SYSTEMS, METHODS AND COMPOSITIONS FOR SEQUENCE MANIPULATION - The invention provides for systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for selecting specific cells by introducing precise mutations utilizing the CRISPR/Cas system. | 06-26-2014 |
20140170753 | CRISPR-CAS SYSTEMS AND METHODS FOR ALTERING EXPRESSION OF GENE PRODUCTS - The invention provides for systems, methods, and compositions for altering expression of target gene sequences and related gene products. Provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for utilizing the CRISPR-Cas system. | 06-19-2014 |
20140107141 | KINASE INHIBITORS AND METHODS OF USE THEREOF - The present invention provides compounds of formula I, pharmaceutically acceptable salts thereof, and pharmaceutical compositions thereof. Compounds of the present invention are useful for inhibiting kinase (e.g., GSK3 (e.g., GSK3α or GSK3β) or CK1) activity. The present invention further provides methods of using the compounds described herein for treating kinase-mediated disorders, such as neurological diseases, psychriatic disorders, metabolic disorders, and cancer. | 04-17-2014 |
20140100223 | COMPOUNDS AND METHODS FOR THE TREATMENT OF ISOCITRATE DEHYDROGENASE RELATED DISEASES - The invention relates to compounds of Formula I or a pharmaceutically acceptable salt, ester or prodrug thereof: | 04-10-2014 |
20140045860 | SMALL MOLECULE INHIBITORS FOR TREATING PARASITIC INFECTIONS - The invention relates to a compound of Formula I or a pharmaceutically acceptable ester, salt, prodrug or metabolite thereof; | 02-13-2014 |
20140031241 | PAIRED END BEAD AMPLIFICATION AND HIGH THROUGHPUT SEQUENCING - The present invention is related to genomic nucleotide sequencing. In particular, the invention describes a paired end sequencing method that enables the sequencing of unique read pairs by co-localizing both 5′ ends on a single emulsion polymerase chain reaction bead. The method may use a customized forked adaptor primer pair that is compatible with massively parallel sequencing techniques. The compositions and methods disclosed herein contemplate sequencing complex genomes, amplified genomic regions, as well as detecting chromosomal structural rearrangements. | 01-30-2014 |
20130331411 | COMPOUNDS AND METHODS FOR TREATING DISEASES MEDIATED BY PROTEASE ACTIVATED RECEPTORS - The invention relates to the use of a compound of Formula I for the treatment of protease-activated receptor mediated diseases by the administration of a compound of Formula I or a prodrug or metabolite thereof. | 12-12-2013 |
20130323744 | Signatures and Determinants Associated with Cancer and Methods of Use Thereof - The present invention provides methods of detecting cancer using biomarkers. | 12-05-2013 |
20130317043 | Compounds and Methods for Treating Autoimmune Diseases - The invention relates to a compound of Formula I: | 11-28-2013 |
20130260376 | Prediction of and Monitoring Cancer Therapy Response Based on Gene Expression Profiling - The invention utilizes gene expression profiles in methods of predicting the likelihood that a patient's cancer will respond to standard-of-care therapy. Also provided are methods of identifying therapeutic agents that target cancer stem cells or epithelial cancers that have undergone an epithelial to mesenchymal transition using such gene expression profiles. | 10-03-2013 |
20130230857 | HYBRID SELECTION USING GENOME-WIDE BAITS FOR SELECTIVE GENOME ENRICHMENT IN MIXED SAMPLES - The present invention provides methods for sequencing and genotyping of DNA useful for analysis of samples in which the target DNA represents a small portion (e.g., 10-1000-fold less) that a contaminating DNA source. Accordingly, the methods described herein are useful for sequencing or genotyping pathogen DNA, such as malaria DNA, in clinical samples taken from infected subjects. | 09-05-2013 |
20130150450 | NOVEL SOLID FORMS OF TACEDINALINE - Novel solid forms of tacedinaline (4-(acetylamino)-N-(2-aminophenyl)benzamide), including crystalline tacedinaline Forms A, B, and D, a novel crystalline tacedinaline TFA salt, and amorphous tacedinaline, are disclosed. Pharmaceutical compositions comprising crystalline tacedinaline Forms A, B, and D, the novel crystalline tacedinaline TFA salt, and/or amorphous tacedinaline, and methods of treating various conditions by administering those novel solid forms, are also disclosed. | 06-13-2013 |
20130149781 | Nucleotide-Specific Recognition Sequences For Designer TAL Effectors - The invention relates to methods of altering expression of a genomic locus of interest or specifically targeting a genomic locus of interest in an animal cell, which may involve contacting the genomic locus with a non-naturally occurring or engineered composition that includes a deoxyribonucleic acid (DNA) binding polypeptide having a N-terminal capping region, a DNA binding domain comprising at least five or more Transcription activator-like effector (TALE) monomers and at least one or more half-monomers specifically ordered to target the genomic locus of interest, and a C-terminal capping region, wherein the polypeptide includes at least one or more effector domains, and wherein the polypeptide is encoded by and translated from a codon optimized nucleic acid molecule so that the polypeptide preferentially binds to the DNA of the genomic locus. | 06-13-2013 |
20130149780 | Nucleotide-Specific Recognition Sequences For Designer TAL Effectors - The invention relates to methods of altering expression of a genomic locus of interest or specifically targeting a genomic locus of interest in an animal cell, which may involve contacting the genomic locus with a non-naturally occurring or engineered composition that includes a deoxyribonucleic acid (DNA) binding polypeptide having a N-terminal capping region, a DNA binding domain comprising at least five or more Transcription activator-like effector (TALE) monomers and at least one or more half-monomers specifically ordered to target the genomic locus of interest, and a C-terminal capping region, wherein the polypeptide includes at least one or more effector domains, and wherein the polypeptide is encoded by and translated from a codon optimized nucleic acid molecule so that the polypeptide preferentially binds to the DNA of the genomic locus. | 06-13-2013 |
20130137161 | Nucleotide-Specific Recognition Sequences For Designer TAL Effectors - The invention relates to methods of altering expression of a genomic locus of interest or specifically targeting a genomic locus of interest in an animal cell, which may involve contacting the genomic locus with a non-naturally occurring or engineered composition that includes a deoxyribonucleic acid (DNA) binding polypeptide having a N-terminal capping region, a DNA binding domain comprising at least five or more Transcription activator-like effector (TALE) monomers and at least one or more half-monomers specifically ordered to target the genomic locus of interest, and a C-terminal capping region, wherein the polypeptide includes at least one or more effector domains, and wherein the polypeptide is encoded by and translated from a codon optimized nucleic acid molecule so that the polypeptide preferentially binds to the DNA of the genomic locus. | 05-30-2013 |
20130137160 | Nucleotide-Specific Recognition Sequences For Designer TAL Effectors - The invention relates to methods of altering expression of a genomic locus of interest or specifically targeting a genomic locus of interest in an animal cell, which may involve contacting the genomic locus with a non-naturally occurring or engineered composition that includes a deoxyribonucleic acid (DNA) binding polypeptide having a N-terminal capping region, a DNA binding domain comprising at least five or more Transcription activator-like effector (TALE) monomers and at least one or more half-monomers specifically ordered to target the genomic locus of interest, and a C-terminal capping region, wherein the polypeptide includes at least one or more effector domains, and wherein the polypeptide is encoded by and translated from a codon optimized nucleic acid molecule so that the polypeptide preferentially binds to the DNA of the genomic locus. | 05-30-2013 |
20130102677 | NOVEL SOLID FORMS OF TACEDINALINE - Novel solid forms of tacedinaline (4-(acetylamino)-N-(2-aminophenyl)benzamide), including crystalline tacedinaline Forms A, B, and D, a novel crystalline tacedinaline TFA salt, and amorphous tacedinaline, are disclosed. Pharmaceutical compositions comprising crystalline tacedinaline Forms A, B, and D, the novel crystalline tacedinaline TFA salt, and/or amorphous tacedinaline, and methods of treating various conditions by administering those novel solid forms, are also disclosed. | 04-25-2013 |
20130018007 | Modified Saponins for the Treatment of Fungal Infections - Methods of treating a fungal infection in a subject, the method comprising administering to the subject a modified saponin. | 01-17-2013 |
20120214821 | Lipidomic Biomarkers of Diabetes - The invention, in some aspects, relates to methods for predicting a subject's risk of developing a glucose-related metabolic disorder, e.g., diabetes. In some aspects, the invention relates to methods for selecting and monitoring a treatment for a glucose-related metabolic disorder, e.g., diabetes. | 08-23-2012 |