NeurosciencebioRxivPreprint
CRISPR-Cas9 targeted deletion of the C9orf72 repeat expansion mutation corrects cellular phenotypes in patient-derived iPS cells
Mochtar Pribadi, Zhongan Yang, Tanya S. Kim…·Giovanni Coppola
CRISPR-Cas9 removes the C9orf72 repeat expansion in iPS cells, reducing RNA foci and promoter hypermethylation without affecting C9orf72 expression.
DOI 10.1101/051193