Bodywide skipping of exons 45–55 in dystrophic mdx52 mice by systemic antisense delivery

Duchenne muscular dystrophy (DMD), the commonest form of muscular dystrophy, is caused by lack of dystrophin. One of the most promising therapeutic approaches is antisense-mediated elimination of frame-disrupting mutations by exon skipping. However, this approach faces two major hurdles: limited app...

Description complète

Enregistré dans:
Détails bibliographiques
Auteurs principaux: Aoki, Yoshitsugu, Yokota, Toshifumi, Nagata, Tetsuya, Nakamura, Akinori, Tanihata, Jun, Saito, Takashi, Duguez, Stephanie M. R., Nagaraju, Kanneboyina, Hoffman, Eric P., Partridge, Terence, Takeda, Shin'ichi
Format: Artigo
Langue:Inglês
Publié: National Academy of Sciences 2012
Sujets:
Biological Sciences
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC3427064/
https://ncbi.nlm.nih.gov/pubmed/22869723
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1204638109
Tags: Ajouter un tag
Pas de tags, Soyez le premier à ajouter un tag!

Documents similaires