In vivo gene editing in dystrophic mouse muscle and muscle stem cells

Science56.90
Volume: 351, Issue: 6271, Pages: 407 - 411
Published: Jan 22, 2016
Abstract
Frame-disrupting mutations in the DMD gene, encoding dystrophin, compromise myofiber integrity and drive muscle deterioration in Duchenne muscular dystrophy (DMD). Removing one or more exons from the mutated transcript can produce an in-frame mRNA and a truncated, but still functional, protein. In this study, we developed and tested a direct gene-editing approach to induce exon deletion and recover dystrophin expression in the mdx mouse model of...
Paper Details
Title
In vivo gene editing in dystrophic mouse muscle and muscle stem cells
Published Date
Jan 22, 2016
Journal
Volume
351
Issue
6271
Pages
407 - 411
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