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

@article{Tabebordbar2016InVG,
  title={In vivo gene editing in dystrophic mouse muscle and muscle stem cells},
  author={Mohammadsharif Tabebordbar and Kexian Zhu and Jason K. W. Cheng and Wei Leong Chew and Jeffrey J Widrick and Winston X. Yan and Claire C. Maesner and Elizabeth Y. Wu and Ru Xiao and Fei Ann Ran and Le Chi Cong and Feng Zhang and Luk H Vandenberghe and George M Church and Amy J. Wagers},
  journal={Science},
  year={2016},
  volume={351},
  pages={407-411}
}
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 DMD. Delivery by adeno-associated virus (AAV) of… CONTINUE READING
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