CRISPR for Neuromuscular Disorders: Gene Editing and Beyond-Reference-Cited by-同舟云学术

CRISPR for Neuromuscular Disorders: Gene Editing and Beyond

Published:2019-09-01 Issue:5 Volume:34 Page:341-353
ISSN:1548-9213
Container-title:Physiology
language:en
Short-container-title:Physiology

Author:

Young Courtney S.¹²³,Pyle April D.²³⁴,Spencer Melissa J.¹²³

Affiliation:

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.

Funder

HHS | NIH | National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Publisher

American Physiological Society

Subject

Physiology

Link

https://www.physiology.org/doi/pdf/10.1152/physiol.00012.2019

Reference141 articles.

1. Therapeutic exon skipping for dysferlinopathies?

2. The CRISPR tool kit for genome editing and beyond

3. CRISPR/Cas9-Induced (CTG⋅CAG) n Repeat Instability in the Myotonic Dystrophy Type 1 Locus: Implications for Therapeutic Genome Editing