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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Vydáno v:Mol Ther
Hlavní autoři: 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.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2018
Témata:
Original Article
On-line přístup: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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