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Application filed by Res Institute At Nationwide Children’S HospitalfiledCriticalRes Institute At Nationwide Children’S Hospital
Publication of AR118192A1publicationCriticalpatent/AR118192A1/en
Medicines Containing Material From Animals Or Micro-Organisms
(AREA)
Abstract
Se describen en el presente documento métodos para tratar la distrofia muscular que comprenden administrar un vector recombinante AAV (VAAr) scAAVrh74.MHCK7.hSGCB, métodos para expresar el gen del b-sarcoglicano en un paciente, composiciones farmacéuticas que comprenden el VAAr, y métodos para generar el VAAr.Described herein are methods for treating muscular dystrophy that comprise administering a recombinant AAV vector (AAVr) scAAVrh74.MHCK7.hSGCB, methods for expressing the b-sarcoglycan gene in a patient, pharmaceutical compositions comprising rAVa, and methods to generate the VAAr.
ARP200100520A2019-02-262020-02-26
ADMINISTRATION OF b-SARCOGLICAN WITH A VECTOR OF ADENO-ASSOCIATED VIRUS AND TREATMENT OF MUSCULAR DYSTROPHY
AR118192A1
(en)