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...

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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2017
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5316861/
https://www.ncbi.nlm.nih.gov/pubmed/28195574
http://dx.doi.org/10.1038/ncomms14454

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