Efficacy and safety of a SOD1-targeting artificial miRNA delivered by AAV9 in mice are impacted by miRNA scaffold selection.
ALS
MT: oligonucleotides: therapies
RNAi
SOD1
adeno-associated virus
gene therapy
preclinical
primary miRNA scaffold
Journal
Molecular therapy. Nucleic acids
ISSN: 2162-2531
Titre abrégé: Mol Ther Nucleic Acids
Pays: United States
ID NLM: 101581621
Informations de publication
Date de publication:
12 Dec 2023
12 Dec 2023
Historique:
received:
27
09
2022
accepted:
12
10
2023
medline:
6
11
2023
pubmed:
6
11
2023
entrez:
6
11
2023
Statut:
epublish
Résumé
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 into the endogenous miRNA pathway, but it is unclear whether different primary miRNA (pri-miRNA) scaffolds impact the potency and safety profile of the expressed amiR
Identifiants
pubmed: 37928442
doi: 10.1016/j.omtn.2023.102057
pii: S2162-2531(23)00275-5
pmc: PMC10622307
doi:
Types de publication
Journal Article
Langues
eng
Pagination
102057Informations de copyright
© 2023 The Author(s).
Déclaration de conflit d'intérêts
M.H. is a paid advisor from Charter Preclinical Services. All other authors are paid employees at Biogen.
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