96904 ⎘
Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used; Muscular dystrophy Duchenne dystrophy
ENGINEERED NUCLEIC ACID REGULATORY ELEMENTS AND METHODS AND USES THEREOF
#2COMPOSITIONS AND METHODS FOR TREATING DUCHENNE MUSCULAR DYSTROPHY
#3RECOMBINANT AAV VECTORS FOR TREATING MUSCULAR DYSTROPHY
#4ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF MICRO-DYSTROPHIN TO TREAT MUSCULAR DYSTROPHY
#5A BIOENGINEERED AAV9 VECTOR CARRYING OPTIMIZED TRANSGENE FOR DUCHENNE MUSCULAR DYSTROPHY GENE THERAPY AND METHOD THEREOF
#6MICRO-DYSTROPHINS AND RELATED METHODS OF USE
#7GENE-BASED MEDICINES AND CELLULAR THERAPY FOR DISEASE
#8METHODS OF MANUFACTURING MICRODYSTROPHIN GENE THERAPY CONSTRUCTS
#9TREATMENT OF DYSTROPHINOPATHIES WITH MICRODYSTROPHIN GENE THERAPY CONSTRUCTS
#10MICRODYSTROPHIN NUCLEIC ACID GENE THERAPY CONSTRUCTS AND USES THEREOF
#11MICRO-DYSTROPHIN FOR HEART PROTECTION
#12ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
#13MODIFIED MUSCLE-SPECIFIC PROMOTERS
#14ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF MICRO-DYSTROPHIN TO TREAT MUSCULAR DYSTROPHY
#15THERAPEUTIC APPLICATIONS OF CPF1-BASED GENOME EDITING
#16TRANSGENIC EXPRESSION CASSETTE FOR TREATING MUSCULAR DYSTROPHY
#17PRODUCTION OF RECOMBINANT AAV VECTORS FOR TREATING MUSCULAR DYSTROPHY
#18GENE THERAPY FOR DUCHENNE MUSCULAR DYSTROPHY
#19ACETAMINOPHEN PROTEIN ADDUCTS AND METHODS OF USE THEREOF
#20SULFATED POLYPEPTIDES FOR SYSTEMIC DELIVERY
#21GENE THERAPY FOR DUCHENNE MUSCULAR DYSTROPHY
#22Methods and Compositions for Modifying a Mutant Dystrophin Gene in a Cell's Genome
#23COMPOSITIONS AND METHODS OF USE THEREOF FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
#24Optimized mini-dystrophin genes and expression cassettes and their use
#25Nucleic Acid-Based Therapy of Muscular Dystrophies
#26TROPHIN GENOME EDITING FOR TREATING DUCHENNE MUSCULAR DYSTROPHY (DMD)
#27DUCHENNE MUSCULAR DYSTROPHY-RELATED EXONIC SPLICING ENHANCER, sgRNA AND GENE EDITING TOOL, AND APPLICATIONS
#28A NOVEL MUSCLE-SPECIFIC PROMOTER
#29MICRODYSTROPHIN GENE THERAPY CONSTRUCTS AND USES THEREOF
#30ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF MICRO-DYSTROPHIN TO TREAT MUSCULAR DYSTROPHY
#31Recombinant Adeno-Associated Virus Delivery of Exon 2-Targeted U7SNRNA Polynucleotide Constructs
#32TREATMENT OF DISEASES CAUSED BY FRAME SHIFT MUTATIONS
#33Adeno-Associated Virus Vector Delivery of Microrna-29 to Treat Muscular Dystrophy
#34PRODUCTION OF LARGE-SIZED QUASIDYSTROPHINS USING OVERLAPPING AAV VECTORS
#35CRISPR/CAS-BASED GENOME EDITING COMPOSITION FOR RESTORING DYSTROPHIN FUNCTION
#36CRISPR/CAS-BASED BASE EDITING COMPOSITION FOR RESTORING DYSTROPHIN FUNCTION
#37ONE-STEP GENE THERAPY FOR DUCHENNE MUSCULAR DYSTROPHY VIA GENE REPLACEMENT AND ANTI-INFLAMMATION
#38COMPOSITIONS COMPRISING THE PROPEPTIDE OF LYSYL OXIDASE AND USES THEREOF
#39ACTRIIB PROTEINS AND VARIANTS AND USES THEREFORE RELATING TO UTROPHIN INDUCTION FOR MUSCULAR DYSTROPHY THERAPY
#40COMPOSITIONS AND METHODS FOR TREATING DUCHENNE MUSCULAR DYSTROPHY
#41HINGES 1 AND/OR 4 MODIFIED DYSTROPHINS FOR DYSTROPHINOPATHY THERAPY
#42Genetic correction of mutated genes
#43RNA-GUIDED GENE EDITING AND GENE REGULATION
#44RNA-GUIDED GENE EDITING AND GENE REGULATION
#45Optimized mini-dystrophin genes and expression cassettes and their use
#46Nucleic Acid-Based Therapy of Muscular Dystrophies
#47COMPOSITIONS AND METHODS OF USE THEREOF FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
#48Micro-dystrophins and related methods of use
#49NOVEL MICRO-DYSTROPHINS AND RELATED METHODS OF USE
#50DMD REPORTER MODELS CONTAINING HUMANIZED DUCHENNE MUSCULAR DYSTROPHY MUTATIONS
#51OPTIMIZED STRATEGY FOR EXON SKIPPING MODIFICATIONS USING CRISPR/CAS9 WITH TRIPLE GUIDE SEQUENCES
#52Acetaminophen adducts and methods of use thereof
#53Recombinant adeno-associated virus delivery of exon 2-targeted U7SNRNA polynucleotide constructs
#54METHODS OF MODIFYING THE DYSTROPHIN GENE AND RESTORING DYSTROPHIN EXPRESSION AND USES THEREOF
#55Therapeutic applications of CPF1-based genome editing
#56Adeno-associated virus vector delivery of microrna-29 to treat muscular dystrophy
#57Adeno-associated virus vector delivery of microRNA-29 and micro-dystrophin to treat muscular dystrophy
#58Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy
#59Methods and compositions for modifying a mutant dystrophin gene in a cell's genome
#60Recombinant Follistatin-FC Fusion Proteins and Use in Treating Duchenne Muscular Dystrophy
#61Production of large-sized microdystrophins in an AAV-based vector configuration
#62RNA-guided gene editing and gene regulation
#63Follistatin in treating duchenne muscular dystrophy
#64MODIFICATION OF THE DYSTROPHIN GENE AND USES THEREOF
#65Micro-dystrophins and related methods of use
#66Microdystrophin peptides and methods for treating muscular dystrophy using the same
#67Methods of increasing sarcolemmal utrophin
#68Genetic correction of mutated genes
#69COMPOSITIONS AND METHODS OF TREATING MUSCULAR DYSTROPHY
#70Compositions and methods for treatment of muscular dystrophy
#71Acetaminophen adducts and methods of use thereof
#72Compositions and methods for treatment of muscular dystrophy
#73RNA-guided gene editing and gene regulation
#74Follistatin in treating duchenne muscular dystrophy
#75Recombinant adeno-associated virus delivery of exon 2-targeted U7SNRNA polynucleotide constructs
#76Methods for enhancing utrophin production via inhibition of microRNA
#77Chimeric dystrophin-VSV-G protein to treat dystrophinopathies
#78WNT7A compositions and method of using the same
#79ActRIIB proteins and variants and uses therefore relating to utrophin induction for muscular dystrophy therapy
#80MICRO-UTROPHIN POLYPEPTIDES AND METHODS
#81Microdystrophin peptides and methods for treating muscular dystrophy using the same
#82Animal models of duchenne muscular dystrophy
#83Gene expression control DNA element and associated protein
#84Prophylactic or ameliorating agent for genetic diseases
#85Nucleic acid molecules and methods for exchanging exon(s) by transsplicing
#86NUCLEIC ACID MOLECULES AND METHODS FOR EXCHANGING EXON(S) BY TRANSSPLICING
#87Methods and materials for producing transgenic artiodactyls
#88Methods and Compositions for Treatment of Muscular Dystrophy
#89Methods for enhancing utrophin production via inhibition of microRNA
#90METHODS AND MATERIALS FOR PRODUCING TRANSGENIC ARTIODACTYLS
#91ActRIIB proteins and variants and uses therefore relating to utrophin induction for muscular dystrophy therapy
#92TAT-utrophin as a protein therapy for dystrophinopathies
#93USE OF GENE VARIANTS OF THE HUMAN MEIS1, BTBD9, MAP2K5, LBXCOR1, PTPRD OR A2BP1 GENE FOR DIAGNOSTIC AND THERAPEUTIC APPROACHES TO RESTLESS LEGS SYNDROME (RLS)
#94TAT-utrophin as a protein therapy for dystrophinopathies
#95Synthetic mini/micro-dystrophin genes to restore nNOS to the sarcolemma
#96Microutrophin and uses thereof
#97Nucleic acid sequences encoding peptides with utrophin spectrin-like repeats
#98Gene expression control DNA element and associated protein
#99Retinal dystrophin transgene and methods of use thereof
#100Drop1, a novel marker for carcinogenesis
#101Methods of treating muscular dystrophy
#102DNA sequences encoding dystrophin minigenes and methods of use thereof
#103Methods of treatment with mini-dystrophin nucleic acid sequences
#104Genetic vaccines for cancer therapy