Attenuation of Heparan Sulfate Proteoglycan Binding Enhances In Vivo Transduction of Human Primary Hepatocytes with AAV2

Use of the prototypical adeno-associated virus type 2 (AAV2) capsid delivered unexpectedly modest efficacy in an early liver-targeted gene therapy trial for hemophilia B. This result is consistent with subsequent data generated in chimeric mouse-human livers showing that the AAV2 capsid transduces p...

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Udgivet i:Mol Ther Methods Clin Dev
Main Authors: Cabanes-Creus, Marti, Westhaus, Adrian, Navarro, Renina Gale, Baltazar, Grober, Zhu, Erhua, Amaya, Anais K., Liao, Sophia H.Y., Scott, Suzanne, Sallard, Erwan, Dilworth, Kimberley L., Rybicki, Arkadiusz, Drouyer, Matthieu, Hallwirth, Claus V., Bennett, Antonette, Santilli, Giorgia, Thrasher, Adrian J., Agbandje-McKenna, Mavis, Alexander, Ian E., Lisowski, Leszek
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2020
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7260615/
https://ncbi.nlm.nih.gov/pubmed/32490035
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.05.004
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