Loading...
Skipping Multiple Exons to Treat DMD—Promises and Challenges
Duchenne muscular dystrophy (DMD) is a lethal disorder caused by mutations in the DMD gene. Antisense-mediated exon-skipping is a promising therapeutic strategy that makes use of synthetic nucleic acids to skip frame-disrupting exon(s) and allows for short but functional protein expression by restor...
Saved in:
| Published in: | Biomedicines |
|---|---|
| Main Authors: | , , |
| Format: | Artigo |
| Language: | Inglês |
| Published: |
MDPI
2018
|
| Subjects: | |
| Online Access: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5874658/ https://ncbi.nlm.nih.gov/pubmed/29301272 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/biomedicines6010001 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|