Next-generation muscle-directed gene therapy by in silico vector design
Adeno-associated viral vectors (AAV) are being developed for gene therapy of skeletal muscle, but it is a challenge to achieve robust gene expression. Here, the authors identify muscle-specific cisregulatory elements that lead to a substantial increase in micro-dystrophin and follistatin expression,...
Enregistré dans:
| Auteurs principaux: | , , , , , , , , , , , , , , , , , , |
|---|---|
| Format: | article |
| Langue: | EN |
| Publié: |
Nature Portfolio
2019
|
| Sujets: | |
| Accès en ligne: | https://doaj.org/article/c5d6b54327424a9fb828402310c7a87f |
| Tags: |
Ajouter un tag
Pas de tags, Soyez le premier à ajouter un tag!
|