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Article

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

16 Oct 2023

Abstract excerpt

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 and mitigate disease progression. Primary microRNA (pri-miRNA) scaffolds are used in amiRs to shuttle a hairpin RNA...

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PMID: 37928442
DOI: 10.1016/j.omtn.2023.102057

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