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

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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Bibliographic Details
Main Authors: Johannes Lengler, Sogue Coulibaly, Bernadette Gruber, Reinhard Ilk, Josef Mayrhofer, Friedrich Scheiflinger, Werner Hoellriegl, Falko G. Falkner, Hanspeter Rottensteiner
Format: Article
Language:English
Published: Elsevier 2020-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
adeno-associated virus
gene therapy
biopotency
coagulation factor IX
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120300437

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http://www.sciencedirect.com/science/article/pii/S2329050120300437

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