In-Depth Characterization of a Mifepristone-Regulated Expression System for AAV5-Mediated Gene Therapy in the Liver

Gene therapy is being developed for the treatment of inherited diseases, whereby a therapeutic gene is continuously expressed in patients after delivery via viral vectors such as adeno-associated virus (AAV). Depending on the transgene, there could be a limited therapeutic window, and regulating tim...

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Detalles Bibliográficos
Autores principales: Liefhebber, Jolanda M., Martier, Raygene, Van der Zon, Tom, Keskin, Sonay, Huseinovic, Angelina, Lubelski, Jacek, Blits, Bas, Petry, Harald, Konstantinova, Pavlina
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6551379/
https://www.ncbi.nlm.nih.gov/pubmed/31194088
http://dx.doi.org/10.1016/j.omtm.2019.05.002

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6551379/
https://www.ncbi.nlm.nih.gov/pubmed/31194088
http://dx.doi.org/10.1016/j.omtm.2019.05.002

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