Gene therapy for limb-girdle muscular dystrophy type 2C

摘要

The present disclosure treats a subject suffering from a gene therapy vector containing a polynucleotide encoding γ-sarcoglycan (SGCG), such as an AAV vector, and muscular dystrophy, such as muscular dystrophy type 2C (LGMD2C). To be concerned with methods of using such gene therapy vectors. In one aspect, a method of treating γ-sarcoglycan dysfunction in a subject is provided, the method comprising administering to the subject a therapeutically effective amount of a recombinant adeno-associated virus (AAV) vector, said rAAV. The vector comprises a gene expression cassette containing a polynucleotide sequence encoding a γ-sarcoglycan under transcriptional control of the promoter, the cassette flanked by one or more AAV inverted terminal repeats.