Development of cell lines with increased susceptibility to diverse adeno-associated viral vectors to enable in vitro potency assays

Vectors based on adeno-associated viruses (AAVs) are promising therapeutic modalities used in gene therapy. Robust cell-based assays that demonstrate and quantify the potency of AAV vectors in expressing their transgene are needed for clinical development. However, many AAV clinical serotypes poorly...

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Bibliographic Details
Main Authors: James Zengel, Emma S. Esterman, Anitha Ponnuswami, Nicholas R. Wall, Jan E. Carette
Format: Article
Language:English
Published: Elsevier 2025-03-01
Series:Molecular Therapy: Methods & Clinical Development
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Online Access:http://www.sciencedirect.com/science/article/pii/S2329050125000117
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