NeuroscienceHuman Molecular GeneticsPeer-reviewed
Correction of amyotrophic lateral sclerosis related phenotypes in induced pluripotent stem cell-derived motor neurons carrying a hexanucleotide expansion mutation in C9orf72 by CRISPR/Cas9 genome editing using homology-directed repair
Nidaa A. Ababneh, Jakub Scaber, Rowan Flynn…·Kevin Talbot
This study corrects a C9orf72 hexanucleotide expansion in iPSC-derived motor neurons using CRISPR/Cas9 and HDR, restoring normal gene expression and eliminating pathological phenotypes.
75 citations