GeneticsFrontiers in Cell and Developmental BiologyPeer-reviewed
Allele-specific gene editing rescues pathology in a human model of Charcot-Marie-Tooth disease type 2E
Carissa M. Feliciano, Kenneth Kun-yu Wu, Hannah L. Watry…·Luke M. Judge
This paper demonstrates allele-specific CRISPR gene editing in a human CMT2E model rescues disease pathology by inactivating the mutant NEFL allele.
25 citations