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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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Bibliografische gegevens
Gepubliceerd in:Mol Ther
Hoofdauteurs: 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.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: American Society of Gene & Cell Therapy 2018
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Online toegang: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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