Work

730. Permanent Correction of Diverse Muscular Dystrophy Mutations in Human Cardiomyocytes by Myoediting

Year: 2016

Type: article

Abstract: Dilated cardiomyopathy (DCM) is one of the most common lethal features of Duchenne muscular dystrophy (DMD), caused by diverse mutations in the X-linked dystrophin gene (DMD). Without dystrophin, a large cytoskeletal protein, muscles degenerate, causing myopathy. Although several gene therapies have been tested, there is no curative treatment so far. In 2014, we first used CRISPR/Cas9-mediated genome editing (termed Myoediting) to correct the mutation in the germ line of mdx mice, the mouse model of DMD [Long et al.

Source: Molecular Therapy

DOI: 10.1016/s1525-0016(16)33538-9