CRISPR for Neuromuscular Disorders: Gene Editing and Beyond.
- Author(s): Young, Courtney S
- Pyle, April D
- Spencer, Melissa J
- et al.
Published Web Locationhttps://doi.org/10.1152/physiol.00012.2019
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.