Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy

Gene replacement therapies utilizing adeno-associated viral (AAV) vectors hold great promise for treating Duchenne muscular dystrophy (DMD). A related approach uses AAV vectors to edit specific regions of the DMD gene using CRISPR/Cas9. Here we develop multiple approaches for editing the mutation in...

ver descrição completa

Na minha lista:
Detalhes bibliográficos
Publicado no:Nat Commun
Main Authors: Bengtsson, Niclas E., Hall, John K., Odom, Guy L., Phelps, Michael P., Andrus, Colin R., Hawkins, R. David, Hauschka, Stephen D., Chamberlain, Joel R., Chamberlain, Jeffrey S.
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2017
Assuntos:
Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5316861/
https://ncbi.nlm.nih.gov/pubmed/28195574
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/ncomms14454
Tags: Adicionar Tag
Sem tags, seja o primeiro a adicionar uma tag!

Registos relacionados