Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy
CRISPR/Cas9-mediated gene editing is an emerging strategy to treat Duchenne muscular dystrophy. Here the authors develop multiple CRISPR/Cas9-based approaches to correct different dystrophin gene mutations, and show significant restoration of dystrophin expression in skeletal and cardiac muscle in m...
Saved in:
| Main Authors: | , , , , , , , , |
|---|---|
| Format: | article |
| Language: | EN |
| Published: |
Nature Portfolio
2017
|
| Subjects: | |
| Online Access: | https://doaj.org/article/42b84f4d2d7e485cb3c0f8f3e7eaf353 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|