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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Bibliographic Details

Published in:	Molecular Therapy: Nucleic Acids
Main Authors:	Shukkwan K. Chen, Zachary C.E. Hawley, Maria I. Zavodszky, Sam Hana, Daniel Ferretti, Branka Grubor, Michael Hawes, Shanqin Xu, Stefan Hamann, Galina Marsh, Patrick Cullen, Ravi Challa, Thomas M. Carlile, Hang Zhang, Wan-Hung Lee, Andrea Peralta, Pete Clarner, Cong Wei, Kathryn Koszka, Feng Gao, Shih-Ching Lo
Format:	Article
Language:	English
Published:	Elsevier 2023-12-01
Subjects:	MT: oligonucleotides: therapies adeno-associated virus ALS SOD1 gene therapy primary miRNA scaffold
Online Access:	http://www.sciencedirect.com/science/article/pii/S2162253123002755