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Application filed by Рисерч Инститьют Эт Нэшнуайд Чилдрен'С ХоспиталfiledCriticalРисерч Инститьют Эт Нэшнуайд Чилдрен'С Хоспитал
Priority claimed from PCT/US2020/019892external-prioritypatent/WO2020176614A1/en
Publication of EA202192346A1publicationCriticalpatent/EA202192346A1/en
Описаны способы лечения мышечной дистрофии, включающие введение вектора рекомбинантного AAV (rAAV) scAAVrh74.MHCK7.hSGCB, способы экспрессии гена бета-саркогликана у пациента, фармацевтические композиции, содержащие rAAV, и способы получения rAAV.Methods for the treatment of muscular dystrophy are described, including the introduction of the recombinant AAV (rAAV) vector scAAVrh74.MHCK7.hSGCB, methods for expressing the beta-sarcoglycan gene in a patient, pharmaceutical compositions containing rAAV, and methods for producing rAAV.
EA202192346A2019-10-042020-02-26
DELIVERY OF -SARCOGLICAN USING A VECTOR BASED ON ADENO ASSOCIATED VIRUS AND TREATMENT OF MUSCULAR DYSTROPHY
EA202192346A1
(en)