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

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 stra...

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Detalles Bibliográficos
Autores principales: Chen, Yi A., Kankel, Mark W., Hana, Sam, Lau, Shukkwan Kelly, Zavodszky, Maria I., McKissick, Olivia, Mastrangelo, Nicole, Dion, Jessica, Wang, Bin, Ferretti, Daniel, Koske, David, Lehman, Sydney, Koszka, Kathryn, McLaughlin, Helen, Liu, Mei, Marshall, Eric, Fabian, Attila J., Cullen, Patrick, Marsh, Galina, Hamann, Stefan, Craft, Michael, Sebalusky, Jennifer, Arnold, H. Moore, Driscoll, Rachelle, Sheehy, Adam, Luo, Yi, Manca, Sonia, Carlile, Thomas, Sun, Chao, Sigrist, Kirsten, McCampbell, Alexander, Henderson, Christopher E., Lo, Shih-Ching
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9713118/
https://www.ncbi.nlm.nih.gov/pubmed/36450833
http://dx.doi.org/10.1038/s41434-022-00375-w

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