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...

Descripción completa

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

Internet

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

Ejemplares similares