Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity

Existing recombinant adeno-associated virus (rAAV) serotypes for delivering in vivo gene therapy treatments for human liver diseases have not yielded combined high-level human hepatocyte transduction and favorable humoral neutralization properties in diverse patient groups. Yet, these combined prope...

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Bibliografiske detaljer
Udgivet i:Mol Ther
Main Authors: Paulk, Nicole K., Pekrun, Katja, Zhu, Erhua, Nygaard, Sean, Li, Bin, Xu, Jianpeng, Chu, Kirk, Leborgne, Christian, Dane, Allison P., Haft, Annelise, Zhang, Yue, Zhang, Feijie, Morton, Chris, Valentine, Marcus B., Davidoff, Andrew M., Nathwani, Amit C., Mingozzi, Federico, Grompe, Markus, Alexander, Ian E., Lisowski, Leszek, Kay, Mark A.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2018
Fag:
Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5763027/
https://ncbi.nlm.nih.gov/pubmed/29055620
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.09.021
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