RECOMBINANT AAV VECTORS FOR TREATING MUSCULAR DYSTROPHY

• Main Authors: RODINO-KLAPAC, Louise, MASON, Stefanie E, MOELLER, Ida H
• Format: Patent
• Language: English; French
• Published: 28.03.2024
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS OR TEST PAPERS THEREFOR; COMPOSITIONS THEREOF; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL ORENZYMOLOGICAL PROCESSES; CULTURE MEDIA; ENZYMOLOGY; HUMAN NECESSITIES; HYGIENE; MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEICACIDS OR MICROORGANISMS; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; ORGANIC CHEMISTRY; PEPTIDES; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROCESSES OF PREPARING SUCH COMPOSITIONS; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS; SPIRITS; VINEGAR; WINE

The present disclosure provides gene therapy vectors, such as recombinant adeno-associated virus (rAAV) for expressing a human micro-dystrophin gene. The present disclosure also provides compositions and methods of using these rAAV to treat muscular dystrophy, such as, e.g., Duchenne Muscular Dystrophy. The present disclosure also provides genotyping a subject's DMD gene to determine if rAAV gene therapy should be contraindicated. La présente invention concerne des vecteurs de thérapie génique, tels qu'un virus adéno-associé recombinant (rAAV) pour exprimer un gène de micro-dystrophine humaine. La présente invention concerne également des compositions et des procédés d'utilisation de ces rAAV pour traiter la dystrophie musculaire, telle que, par exemple, la dystrophie musculaire de Duchenne. La présente invention concerne également le génotypage du gène DMD d'un sujet pour déterminer si une thérapie génique rAAV est contre-indiquée.