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Open Access Publications from the University of California

CRISPR for Neuromuscular Disorders: Gene Editing and Beyond.

  • Author(s): Young, Courtney S
  • Pyle, April D
  • Spencer, Melissa J
  • et al.

This is a review describing advances in CRISPR/Cas-mediated therapies for neuromuscular disorders (NMDs). We explore both CRISPR-mediated editing and dead Cas approaches as potential therapeutic strategies for multiple NMDs. Last, therapeutic considerations, including delivery and off-target effects, are also discussed.

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