CRISPR/Cas correction of muscular dystrophies

Yu Zhang, Takahiko Nishiyama, Eric N Olson, Rhonda Bassel-Duby

Research output: Contribution to journalArticlepeer-review

Abstract

Muscular dystrophies are a heterogeneous group of monogenic neuromuscular disorders which lead to progressive muscle loss and degeneration of the musculoskeletal system. The genetic causes of muscular dystrophies are well characterized, but no effective treatments have been developed so far. The discovery and application of the CRISPR/Cas system for genome editing offers a new path for disease treatment with the potential to permanently correct genetic mutations. The post-mitotic and multinucleated features of skeletal muscle provide an ideal target for CRISPR/Cas therapeutic genome editing because correction of a subpopulation of nuclei can provide benefit to the whole myofiber. In this review, we provide an overview of the CRISPR/Cas system and its derivatives in genome editing, proposing potential CRISPR/Cas-based therapies to correct diverse muscular dystrophies, and we discuss challenges for translating CRISPR/Cas genome editing to a viable therapy for permanent correction of muscular dystrophies.

Original languageEnglish (US)
Article number112844
JournalExperimental Cell Research
Volume408
Issue number1
DOIs
StatePublished - Nov 1 2021

Keywords

  • Duchenne muscular dystrophy
  • Facioscapulohumeral muscular dystrophy
  • Genome editing
  • Limb-girdle muscular dystrophy
  • sgRNA
  • Skeletal muscle

ASJC Scopus subject areas

  • Cell Biology

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