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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...

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Αποθηκεύτηκε σε:
Λεπτομέρειες βιβλιογραφικής εγγραφής
Τόπος έκδοσης:Mol Ther Methods Clin Dev
Κύριοι συγγραφείς: 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.
Μορφή: Artigo
Γλώσσα:Inglês
Έκδοση: American Society of Gene & Cell Therapy 2020
Θέματα:
Διαθέσιμο Online: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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