Article
Development of Patient-specific AAV Vectors After Neutralizing Antibody Selection for Enhanced Muscle Gene Transfer.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Feb 2016
Li Chengwen, Wu Shuqing, Albright Blake, Hirsch Matthew, Li Wuping, Tseng Yu-Shan, Agbandje-McKenna Mavis, McPhee Scott, Asokan Aravind, Samulski R Jude
Abstract excerpt
A major hindrance in gene therapy trials with adeno-associated virus (AAV) vectors is the presence of neutralizing antibodies (NAbs) that inhibit AAV transduction. In this study, we used directed evolution techniques in vitro and in mouse muscle to select novel NAb escape AAV chimeric capsid mutants in the presence of individual patient serum. AAV mutants isolated in vitro escaped broad patient-specific NAb...
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