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

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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Bibliographic Details
Main Authors: Carissa M. Feliciano, Kenneth Wu, Hannah L. Watry, Chiara B. E. Marley, Gokul N. Ramadoss, Hana Y. Ghanim, Angela Z. Liu, Lyandysha V. Zholudeva, Todd C. McDevitt, Mario A. Saporta, Bruce R. Conklin, Luke M. Judge
Format: Article
Language:English
Published: Frontiers Media S.A. 2021-08-01
Series:Frontiers in Cell and Developmental Biology
Subjects:
induced pluripotent stem cells
Charcot-Marie-Tooth
neuropathy
motor neurons
dominant
CRISPR-Cas9
Online Access:https://www.frontiersin.org/articles/10.3389/fcell.2021.723023/full

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