CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice.

  title={CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice.},
  author={Li Xu and Ki Ho Park and Lixia Zhao and Jing Xu and Mona M. El Refaey and Yandi Gao and Hua Zhu and Jianjie Ma and Renzhi Han},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  volume={24 3},
Duchenne muscular dystrophy (DMD) is a degenerative muscle disease caused by genetic mutations that lead to the disruption of dystrophin in muscle fibers. There is no curative treatment for this devastating disease. Clustered regularly interspaced short palindromic repeat/Cas9 (CRISPR/Cas9) has emerged as a powerful tool for genetic manipulation and potential therapy. Here we demonstrate that CRIPSR-mediated genome editing efficiently excised a 23-kb genomic region on the X-chromosome covering… CONTINUE READING
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