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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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Detalhes bibliográficos
Publicado no: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
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2016
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Acesso em linha: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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