Development of AAV Variants with Human Hepatocyte Tropism and Neutralizing Antibody Escape Capacity.
AAV
Nabs
chimeric mice
human hepatocyte
tropism
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:
11 Sep 2020
11 Sep 2020
Historique:
received:
20
02
2020
accepted:
29
05
2020
entrez:
9
7
2020
pubmed:
9
7
2020
medline:
9
7
2020
Statut:
epublish
Résumé
Adeno-associated virus (AAV) vectors have been successfully used in patients with bleeding disorders and blindness. For human liver targeting, two major factors restrict effective AAV transduction after systemic administration of AAV vectors: human hepatocyte tropism and neutralizing antibodies (Nabs). In this study, we attempted to isolate AAV variants with the ability to transduce human hepatocytes and escape Nabs using a directed evolution approach
Identifiants
pubmed: 32637455
doi: 10.1016/j.omtm.2020.06.003
pii: S2329-0501(20)30125-X
pmc: PMC7329936
doi:
Types de publication
Journal Article
Langues
eng
Pagination
259-268Informations de copyright
© 2020 The Authors.
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