Efficacy and safety of a SOD1-targeting artificial miRNA delivered by AAV9 in mice are impacted by miRNA scaffold selection

Toxic gain-of-function mutations in superoxide dismutase 1 (SOD1) contribute to approximately 2%–3% of all amyotrophic lateral sclerosis (ALS) cases. Artificial microRNAs (amiRs) delivered by adeno-associated virus (AAV) have been proposed as a potential treatment option to silence SOD1 expression a...

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Detalles Bibliográficos
Autores principales: Chen, Shukkwan K., Hawley, Zachary C.E., Zavodszky, Maria I., Hana, Sam, Ferretti, Daniel, Grubor, Branka, Hawes, Michael, Xu, Shanqin, Hamann, Stefan, Marsh, Galina, Cullen, Patrick, Challa, Ravi, Carlile, Thomas M., Zhang, Hang, Lee, Wan-Hung, Peralta, Andrea, Clarner, Pete, Wei, Cong, Koszka, Kathryn, Gao, Feng, Lo, Shih-Ching
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10622307/
https://www.ncbi.nlm.nih.gov/pubmed/37928442
http://dx.doi.org/10.1016/j.omtn.2023.102057

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