Myoediting: Toward prevention of muscular dystrophy by therapeutic genome editing

Yu Zhang, Chengzu Long, Rhonda Bassel-Duby, Eric N. Olson

Research output: Contribution to journalReview article

7 Scopus citations

Abstract

Muscular dystrophies represent a large group of genetic disorders that significantly impair quality of life and often progress to premature death. There is no effective treatment for these debilitating diseases. Most therapies, developed to date, focus on alleviating the symptoms or targeting the secondary effects, while the underlying gene mutation is still present in the human genome. The discovery and application of programmable nucleases for site-specific DNA doublestranded breaks provides a powerful tool for precise genome engineering. In particular, the CRISPR/Cas system has revolutionized the genome editing field and is providing a new path for disease treatment by targeting the disease-causing genetic mutations. In this review, we provide a historical overview of genome-editing technologies, summarize the most recent advances, and discuss potential strategies and challenges for permanently correcting genetic mutations that cause muscular dystrophies.

Original languageEnglish (US)
Pages (from-to)1205-1240
Number of pages36
JournalPhysiological Reviews
Volume98
Issue number3
DOIs
StatePublished - Jul 1 2018

ASJC Scopus subject areas

  • Physiology
  • Molecular Biology
  • Physiology (medical)

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