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

Autor: 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
Rok vydání: 2022
Předmět:
Zdroj: Gene Therapy. 30:443-454
ISSN: 1476-5462
0969-7128
DOI: 10.1038/s41434-022-00375-w
Popis: 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 SOD1
Databáze: OpenAIRE
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