Full-Length Dystrophin Restoration via Targeted Exon Addition in DMD-Patient Specific iPSCs and Cardiomyocytes

Duchenne muscular dystrophy (DMD) is the most common fatal muscle disease, with an estimated incidence of 1/3500–1/5000 male births, and it is associated with mutations in the X-linked DMD gene encoding dystrophin, the largest known human gene. There is currently no cure for DMD. The large size of t...

Descripción completa

Detalles Bibliográficos
Autores principales: Xiao, Rou, Zhou, Miaojin, Wang, Peiyun, Zeng, Baitao, Wu, Lingqian, Hu, Zhiqing, Liang, Desheng
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9409156/
https://www.ncbi.nlm.nih.gov/pubmed/36012442
http://dx.doi.org/10.3390/ijms23169176

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9409156/
https://www.ncbi.nlm.nih.gov/pubmed/36012442
http://dx.doi.org/10.3390/ijms23169176

Ejemplares similares