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CRISPR Correction of Duchenne Muscular Dystrophy

Authors
  • Min, Yi-Li
  • Bassel-Duby, Rhonda
  • Olson, Eric N.
Type
Published Article
Journal
Annual Review of Medicine
Publisher
Annual Reviews
Publication Date
Jan 27, 2019
Volume
70
Pages
239–255
Identifiers
DOI: 10.1146/annurev-med-081117-010451
Source
Annual Reviews
Keywords
License
Yellow

Abstract

The ability to efficiently modify the genome using CRISPR technology has rapidly revolutionized biology and genetics and will soon transform medicine. Duchenne muscular dystrophy (DMD) represents one of the first monogenic disorders that has been investigated with respect to CRISPR-mediated correction of causal genetic mutations. DMD results from mutations in the gene encoding dystrophin, a scaffolding protein that maintains the integrity of striated muscles. Thousands of different dystrophin mutations have been identified in DMD patients, who suffer from a loss of ambulation followed by respiratory insufficiency, heart failure, and death by the third decade of life. Using CRISPR to bypass DMD mutations, dystrophin expression has been efficiently restored in human cells and mouse models of DMD. Here, we review recent progress toward the development of possible CRISPR therapies for DMD and highlight opportunities and potential obstacles in attaining this goal.

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