Clinical Considerations for Capsid Choice in the Development of Liver-Targeted AAV-Based Gene Transfer

As gene transfer with adeno-associated virus (AAV) vectors is starting to enter clinical practice, this review examines the impact of vector capsid choice in liver-directed gene transfer for hemophilia. Given that there are multiple clinical trials completed and ongoing in this field, it is importan...

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Bibliographic Details
Main Authors: Steven Pipe, Frank W.G. Leebeek, Valerie Ferreira, Eileen K. Sawyer, John Pasi
Format: Article
Language:English
Published: Elsevier 2019-12-01
Series:Molecular Therapy: Methods & Clinical Development
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050119300956