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Development of an In Vitro Biopotency Assay for an AAV8 Hemophilia B Gene Therapy Vector Suitable for Clinical Product Release

Gene therapy product release requires reliable and consistent demonstration of biopotency. In hemophilia B vectors, this is usually determined in vivo by measuring the plasma levels of the expressed human factor IX (FIX) transgene product in FIX knockout mice. To circumvent this laborious assay, we...

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Detalles Bibliográficos
Autores principales:	Lengler, Johannes, Coulibaly, Sogue, Gruber, Bernadette, Ilk, Reinhard, Mayrhofer, Josef, Scheiflinger, Friedrich, Hoellriegl, Werner, Falkner, Falko G., Rottensteiner, Hanspeter
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/PMC7139127/ https://www.ncbi.nlm.nih.gov/pubmed/32280725 http://dx.doi.org/10.1016/j.omtm.2020.03.013

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