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 Carlile, Chao Sun, Kirsten Sigrist, Alexander McCampbell, Christopher E Henderson, Shih-Ching Lo
CRISPR-based gene editing technology represents a promising approach to deliver therapies for inherited disorders, including amyotrophic lateral sclerosis (ALS). Toxic gain-of-function superoxide dismutase 1 (SOD1) mutations are responsible for ~20% of familial ALS cases. Thus, current clinical strategies to treat SOD1-ALS are designed to lower SOD1 levels. Here, we utilized AAV-PHP.B variants to deliver CRISPR-Cas9 guide RNAs designed to disrupt the human SOD1 (huSOD1) transgene in SOD1G93A mice. A one-time intracerebroventricular injection of AAV...
December 1, 2022: Gene Therapy