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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...

תיאור מלא

שמור ב:
מידע ביבליוגרפי
הוצא לאור ב:Mol Ther Methods Clin Dev
Main Authors: 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.
פורמט: Artigo
שפה:Inglês
יצא לאור: American Society of Gene & Cell Therapy 2020
נושאים:
גישה מקוונת:https://ncbi.nlm.nih.gov/pmc/articles/PMC7327880/
https://ncbi.nlm.nih.gov/pubmed/32637454
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.06.002
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