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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Bibliografske podrobnosti
izdano v: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
Jezik:Inglês
Izdano: Nature Publishing Group 2016
Teme:
Original Article
Online dostop: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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