AAV-shDUX4 provides short-term benefits but limited long-term efficacy in a DUX4 mouse model of FSHD
The aberrant expression of the toxic transcription factor DUX4 in skeletal muscle is a hallmark of facioscapulohumeral muscular dystrophy. Effective therapeutic strategies will likely require the inhibition of DUX4, with adeno-associated virus (AAV)-mediated therapies being among the promising appro...
Saved in:
| Main Authors: | , , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Elsevier
2025-09-01
|
| Series: | Molecular Therapy: Methods & Clinical Development |
| Subjects: | |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2329050125001299 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|