Development of Patient-specific AAV Vectors After Neutralizing Antibody Selection for Enhanced Muscle Gene Transfer

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...

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Bibliografiske detaljer
Udgivet i:Mol Ther
Main Authors: Li, Chengwen, Wu, Shuqing, Albright, Blake, Hirsch, Matthew, Li, Wuping, Tseng, Yu-Shan, Agbandje-McKenna, Mavis, McPhee, Scott, Asokan, Aravind, Samulski, R Jude
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group 2016
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Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4754536/
https://ncbi.nlm.nih.gov/pubmed/26220272
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2015.134
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