Allele-Specific Gene Editing Rescues Pathology in a Human Model of Charcot-Marie-Tooth Disease Type 2E

Many neuromuscular disorders are caused by dominant missense mutations that lead to dominant-negative or gain-of-function pathology. This category of disease is challenging to address via drug treatment or gene augmentation therapy because these strategies may not eliminate the effects of the mutant...

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Detalles Bibliográficos
Autores principales: Feliciano, Carissa M., Wu, Kenneth, Watry, Hannah L., Marley, Chiara B. E., Ramadoss, Gokul N., Ghanim, Hana Y., Liu, Angela Z., Zholudeva, Lyandysha V., McDevitt, Todd C., Saporta, Mario A., Conklin, Bruce R., Judge, Luke M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2021
Materias:
Cell and Developmental Biology
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8415563/
https://www.ncbi.nlm.nih.gov/pubmed/34485306
http://dx.doi.org/10.3389/fcell.2021.723023

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