Correction of Three Prominent Mutations in Mouse and Human Models of Duchenne Muscular Dystrophy by Single-Cut Genome Editing

Duchenne muscular dystrophy (DMD), one of the most common neuromuscular disorders of children, is caused by the absence of dystrophin protein in striated muscle. Deletions of exons 43, 45, and 52 represent mutational “hotspot” regions in the dystrophin gene. We created three new DMD mouse models har...

ver descrição completa

Na minha lista:
Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Min, Yi-Li, Chemello, Francesco, Li, Hui, Rodriguez-Caycedo, Cristina, Sanchez-Ortiz, Efrain, Mireault, Alex A., McAnally, John R., Shelton, John M., Zhang, Yu, Bassel-Duby, Rhonda, Olson, Eric N.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2020
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7474267/
https://ncbi.nlm.nih.gov/pubmed/32892813
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.05.024
Tags: Adicionar Tag
Sem tags, seja o primeiro a adicionar uma tag!

Registos relacionados