A Quantitative In Vitro Potency Assay for Adeno-Associated Virus Vectors Encoding for the UGT1A1 Transgene

Potency assessment of clinical-grade vector lots is crucial to support adeno-associated virus (AAV) vector release and is required for future marketing authorization. We have developed and validated a cell-based, quantitative potency assay that detects both transgenic expression and activity of an A...

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Detalles Bibliográficos
Autores principales: Aronson, Sem J., Bakker, Robert S., Moenis, Sascha, van Dijk, Remco, Bortolussi, Giulia, Collaud, Fanny, Shi, Xiaoxia, Duijst, Suzanne, ten Bloemendaal, Lysbeth, Ronzitti, Giuseppe, Muro, Andrés F., Mingozzi, Federico, Beuers, Ulrich, Bosma, Piter J.
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/PMC7327880/
https://www.ncbi.nlm.nih.gov/pubmed/32637454
http://dx.doi.org/10.1016/j.omtm.2020.06.002

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7327880/
https://www.ncbi.nlm.nih.gov/pubmed/32637454
http://dx.doi.org/10.1016/j.omtm.2020.06.002

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