In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model

Yi A. Chen, Mark W. Kankel, Sam Hana, Shukkwan Kelly Lau, Maria I. Zavodszky, Olivia McKissick, Nicole Mastrangelo, Jessica Dion, Bin Wang, Daniel Ferretti, David Koske, Sydney Lehman, Kathryn Koszka, Helen McLaughlin, Mei Liu, Eric Marshall, Attila J. Fabian, Patrick Cullen, Galina Marsh, Stefan Hamann, Michael Craft, Jennifer Sebalusky, H. Moore Arnold, Rachelle Driscoll, Adam Sheehy, Yi Luo, Sonia Manca, Thomas M. Carlile, Chao Sun, Kirsten Sigrist, Alexander McCampbell, Christopher E. Henderson, Shih‐Ching Lo