[Report] In vivo gene editing in dystrophic mouse muscle and muscle stem cells

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 DMD. Delivery by adeno-associated virus (AAV) of clustered regularly interspaced short palindromic repeats (CRISPR)–Cas9 endonucleases coupled with paired guide RNAs flanking the mutated Dmd exon23 resulted in excision of intervening DNA and restored the Dmd reading frame in myofibers, cardiomyocytes, and muscle stem cells after local or systemic delivery. AAV-Dmd CRISPR treatment partially recovered muscle functional deficiencies and generated a pool of endogenously corrected myogenic precursors in mdx mouse muscle. Authors: Mohammadsharif Tabebordbar, Kexian Zhu, Jason K. W. Cheng, Wei Leong Chew, Jeffrey J. Widrick, Winston X. Yan, Claire Maesner, Elizabeth Y. Wu, Ru Xiao, F. Ann Ran, Le Cong, Feng Zhang, Luk H. Vandenberghe, George M. Church, Amy J. Wagers
Source: ScienceNOW - Category: Science Authors: Source Type: news