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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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7260615/
https://www.ncbi.nlm.nih.gov/pubmed/32490035
http://dx.doi.org/10.1016/j.omtm.2020.05.004

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7260615/
https://www.ncbi.nlm.nih.gov/pubmed/32490035
http://dx.doi.org/10.1016/j.omtm.2020.05.004

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