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, Andrew G. L. Douglas, Paola Barbagallo, Ana Candalija, Martin R. Turner, David Sims, Ruxandra Dafinca, Sally A. Cowley, 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.
Human Molecular Genetics · Jun 1, 2020Peer-reviewedNeuroscience