AAV gene therapy methods for treating muscular dystrophy

Abstract

muscular dystrophy，DMD)。 The invention described herein provides a method of treatment for a disease in a human in need thereof, comprising administering to the human an rAAV viral particle comprising a polynucleotide encoding a therapeutic gene-of-interest, as described herein. The method can be used for delivering a gene of interest (GOI), such as a microdystrophin-encoding polynucleotide sequence, for the treatment of a disease, e.g., muscular dystrophy such as Duchenne muscular dystrophy (DMD).

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