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, Elliot W. Swartz, Alden Y. Huang, Jason A. Chen, Deepika Dokuru, Jaeyun Baek, Fuying Gao, Andrea T. Fua, Kevin Wojta, Qing Wang, Anna Karydas, Jamie Fong, Ed Lezcano, , Giovanni Coppola
CRISPR-Cas9 removes the C9orf72 repeat expansion in iPS cells, reducing RNA foci and promoter hypermethylation without affecting C9orf72 expression.
bioRxiv · May 2, 2016 · PreprintNeuroscience