Application of in-vitro-cultured primary hepatocytes to evaluate species translatability and AAV transduction mechanisms of action

Application of in-vitro-cultured primary hepatocytes to evaluate species translatability and AAV transduction mechanisms of action

Recombinant adeno-associated virus (AAV) is an effective platform for therapeutic gene transfer; however, tissue-tropism differences between species are a challenge for successful translation of preclinical results to humans. We evaluated the use of in vitro primary hepatocyte cultures to predict in...

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Bibliographic Details
Main Authors: Su Liu, Lisa Razon, Olivia Ritchie, Choong-Ryoul Sihn, Britta Handyside, Geoffrey Berguig, Jill Woloszynek, Lening Zhang, Paul Batty, David Lillicrap, Vishal Agrawal, Christa Cortesio, Kahsay Gebretsadik, Hassibullah Akeefe, Peter Colosi, Benjamin Kim, Stuart Bunting, Sylvia Fong
Format: Article
Language:English
Published: Elsevier 2022-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
AAV vectors
AAV transduction
hepatocytes
in vitro model
valoctocogene roxaparvovec
AAV5-hFVIII-SQ
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050122000730

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http://www.sciencedirect.com/science/article/pii/S2329050122000730

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