CRISPR for Neuromuscular Disorders: Gene Editing and Beyond

Courtney S Young; April D Pyle; Melissa J Spencer

doi:10.1152/physiol.00012.2019

CRISPR for Neuromuscular Disorders: Gene Editing and Beyond

Physiology (Bethesda). 2019 Sep 1;34(5):341-353. doi: 10.1152/physiol.00012.2019.

Authors

Courtney S Young^{1

2

3}, April D Pyle^{2

3

4}, Melissa J Spencer^{1

2

3}

Affiliations

¹ Department of Neurology, University of California, Los Angeles, California.
² Center for Duchenne Muscular Dystrophy at UCLA, University of California, Los Angeles, California.
³ Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, University of California, Los Angeles, California.
⁴ Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, California.

Abstract

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.

Keywords: CRISPR/Cas; gene editing; gene therapy; muscular dystrophy; neuromuscular disorders.

Publication types

Research Support, N.I.H., Extramural
Review

MeSH terms

Animals
Clustered Regularly Interspaced Short Palindromic Repeats / genetics*
Gene Editing / methods
Humans
Neuromuscular Diseases / genetics*

Abstract

Publication types

MeSH terms

Grants and funding