A universal gene correction approach for FKRP-associated dystroglycanopathies to enable autologous cell therapy

Mutations in the fukutin-related protein (FKRP) gene result in a broad spectrum of muscular dystrophy (MD) phenotypes, including the severe Walker-Warburg syndrome (WWS). Here, we develop a gene-editing approach that replaces the entire mutant open reading frame with the wild-type sequence to univer...

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Detalles Bibliográficos
Autores principales: Dhoke, Neha R., Kim, Hyunkee, Selvaraj, Sridhar, Azzag, Karim, Zhou, Haowen, Oliveira, Nelio A.J., Tungtur, Sudheer, Ortiz-Cordero, Carolina, Kiley, James, Lu, Qi Long, Bang, Anne G., Perlingeiro, Rita C.R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8327854/
https://www.ncbi.nlm.nih.gov/pubmed/34260922
http://dx.doi.org/10.1016/j.celrep.2021.109360

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