96903 ⎘
Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used Muscular dystrophy
Sub-classes:GENE THERAPY FOR LIMB-GIRDLE MUSCULAR DYSTROPHY TYPE 2C
#2MICRO-DYSTROPHINS AND RELATED METHODS OF USE
#3MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING DYSTROPHINOPATHIES
#4METHOD FOR EXPRESSING A MUSCLE-SPECIFIC GENE AND CASSETTES FOR SAME
#5MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING DYSTROPHINOPATHIES
#6COMPOSITIONS AND METHODS FOR RECOMBINANT AAV PRODUCTION
#7MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING DYSTROPHINOPATHIES
#8MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING DYSTROPHINOPATHIES
#9ANTIBODY-EVADING VIRUS VECTORS
#10Muscle targeting complexes and uses thereof for treating dystrophinopathies
#11Muscle targeting complexes and uses thereof for treating dystrophinopathies
#12METHODS AND COMPOSITIONS TO SPREAD PROTEIN CARGOES ACROSS MULTI-NUCLEATED CELLS
#13TREATMENT OF MUSCULAR DYSTROPHY
#14C2 Domain Therapeutics and Uses Thereof
#15MODIFIED AAV CAPSIDS AND USES THEREOF
#16RECOMBINANT ADENO-ASSOCIATED VIRUS PRODUCTS AND METHODS FOR TREATING DYSTROGLYCANOPATHIES AND LAMININ-DEFICIENT MUSCULAR DYSTROPHIES
#17Muscle targeting complexes and uses thereof for treating myotonic dystrophy
#18COMPOSITION FOR TREATING DYSFERLINOPATHY
#19Methods of Treating Muscular Dystrophy
#20SYNTHETIC INTERMEMBRANE PROTEOLYSIS RECEPTORS FOR CUSTOM ANTIGEN-INDUCED TRANSCRIPTIONAL REGULATION
#21Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
#22Antibody-evading virus vectors
#23EXON SKIPPING OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY
#24Treatment of muscular dystrophies
#25A HIGH-THROUGHPUT SCREENING METHOD TO DISCOVER OPTIMAL GRNA PAIRS FOR CRISPR-MEDIATED EXON DELETION
#26NOVEL POLYNUCLEOTIDES ENCODING A HUMAN FKRP PROTEIN
#27Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
#28Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
#29Self-Complementary Adeno-Associated Virus Vector and its Use in Treatment of Muscular Dystrophy
#30GENE THERAPY WITH DYSFERLIN DUAL VECTORS
#31OPTIMIZED GENE THERAPY FOR TARGETING MUSCLE IN MUSCLE DISEASES
#32Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
#33EXON SKIPPING OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY
#34Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
#35Recombinant Adeno-Associated Virus Delivery of Alpha-Sarcoglycan Polynucleotides
#36RECOMBINANT VIRUS PRODUCTS AND METHODS FOR INHIBITION OF EXPRESSION OF MYOTILIN
#37ACTRIIB PROTEINS AND VARIANTS AND USES THEREFORE RELATING TO UTROPHIN INDUCTION FOR MUSCULAR DYSTROPHY THERAPY
#38Antibody-evading virus vectors
#39Synthetic polynucleotides encoding a human FKRP protein
#40Exon skipping oligomer conjugates for muscular dystrophy
#41Exon skipping oligomer conjugates for muscular dystrophy
#42Treatment of muscular dystrophies
#43Gene therapy for limb-girdle muscular dystrophy type 2C
#44Micro-dystrophins and related methods of use
#45NOVEL MICRO-DYSTROPHINS AND RELATED METHODS OF USE
#46Truncated dysferlin for treatment of dysferlinopathy
#47Exon skipping oligomer conjugates for muscular dystrophy
#48Exon skipping oligomer conjugates for muscular dystrophy
#49Methods and Compositions for Treating Dystroglycanopathy Disorders
#50Recombinant virus products and methods for inhibition of expression of myotilin
#51Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
#52Methods of treating muscular dystrophy
#53Selective gene therapy expression system
#54Compositions for treating dystroglycanopathy disorders
#55Methods of increasing sarcolemmal utrophin
#56Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
#57Recombinant virus products and methods for inhibition of expression of myotilin
#58Selective gene therapy expression system
#59Nucleic acids encoding FHL1 mutations associated with novel X-linked muscular myopathies and methods of screening a subject
#60METHODS AND COMPOSITIONS RELATED TO THE SMCHD1 GENE
#61WNT7A compositions and method of using the same
#62ActRIIB proteins and variants and uses therefore relating to utrophin induction for muscular dystrophy therapy
#63Recombinant virus products and methods for inhibition of expression of myotilin
#64Prophylactic or ameliorating agent for genetic diseases
#65Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations
#66ActRIIB proteins and variants and uses therefore relating to utrophin induction for muscular dystrophy therapy
#67ADENO-ASSOCIATED VIRAL VECTORS FOR THE EXPRESSION OF DYSFERLIN
#68Methods of screening a subject for mutations associated with novel X-linked muscular myopathies
#69Nucleic acid codifying for a utrophin transcription specific regulating protein, protein codified thereby and uses thereof
#70Protein-coding RNA to correct mitochondrial dysfunction
#71Product and Methods for Diagnosis and Therapy for Cardiac and Skeletal Muscle Disorders
#72Hybrid-AAV vectors to deliver large gene expression cassette
#73Chn-1/Chip-Antagonists For The Treatment Of Muscular Diseases
#74Composition and Methods for the Treatment of Duchene Muscular Dystrophy
#75Product and Methods for Diagnosis and Therapy for Cardiac and Skeletal Muscle Disorders
#76Increasing functional glycosylation of alpha-dystroglycan in the treatment of muscle degeneration
#77Product and methods for diagnosis and therapy for cardiac and skeletal muscle disorders
#78Survival motor neuron gene (SMN2) mRNA constructs for post-transcription regulation