UTROPHIN GENOME EDITING FOR TREATING DUCHENNE MUSCULAR DYSTROPHY (DMD)

摘要

The invention relates to compositions and methods for improving a dystrophic phenotype in a human subject having myopathies, such as Duchenne Muscular Dystrophy (DMD). In one embodiment, the invention relates to compositions comprising an adenoviral vector targeting the let-7c miRNA binding sequence in 3'-UTR genome editing of the utrophin gene and methods of treatment comprising administration thereof.