Development of a one-step RT-ddPCR method to determine the expression and potency of AAV vectors.
AAV gene therapy
assay development
one-step RT-ddPCR
potency assay
vector expression
Journal
Molecular therapy. Methods & clinical development
ISSN: 2329-0501
Titre abrégé: Mol Ther Methods Clin Dev
Pays: United States
ID NLM: 101624857
Informations de publication
Date de publication:
10 Dec 2021
10 Dec 2021
Historique:
received:
26
01
2021
accepted:
07
05
2021
entrez:
30
9
2021
pubmed:
1
10
2021
medline:
1
10
2021
Statut:
epublish
Résumé
Robust assays to quantify adeno-associated virus (AAV) vector expression and potency are essential for gene therapy development. These assays inform the efficacy, safety, and pharmacodynamic profiles of AAV development candidates. Additionally, for gene downregulation strategies such as RNAi, knockdown of endogenous genes reflects the mechanism of action of such development candidates. Therefore, a method to quantify target mRNA repression is necessary for measuring vector potency both
Identifiants
pubmed: 34589555
doi: 10.1016/j.omtm.2021.05.003
pii: S2329-0501(21)00087-5
pmc: PMC8449018
doi:
Types de publication
Journal Article
Langues
eng
Pagination
68-77Informations de copyright
© 2021 The Author(s).
Déclaration de conflit d'intérêts
All authors were employees at Biogen at the time of the studies. The authors declare no competing interests.
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