Superior human hepatocyte transduction with adeno-associated virus vector serotype 7
Abstract
Although therapeutic outcomes have been achieved in hemophilia patients after delivery of clotting factor genes to the liver using adeno-associated virus (AAV) vectors, it is well known that the preclinical results generated from hemophilia animal models have not been directly predictive of successful translation in humans. To address this discrepancy humanized mouse models have recently been used to predict AAV transduction efficiency for human...
Paper Details
Title
Superior human hepatocyte transduction with adeno-associated virus vector serotype 7
Published Date
Sep 30, 2019
Journal
Volume
26
Issue
12
Pages
504 - 514
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