Immortalized MUT MEFs are an excellent tool for developing potency assays to validate novel FRDA therapies. Care needs to be exercised while utilizing these cell lines, as extended passaging results in molecular changes that spontaneously reverse FRDA-like phenotypes without increasing Fxn expression. Based on transcriptome analyses, we identified the Warburg effect as the mechanism allowing cells expressing a minimal level of Fxn to thrive under standard cell culture conditions.
Sunday, October 27, 2024
Design and validation of cell-based potency assays for frataxin supplementation treatments
Design and validation of cell-based potency assays for frataxin supplementation treatments; Shibani Mukherjee, Larisa Pereboeva, Daniel Fil, Achisha Saikia, Jeon Lee, Jixue Li, M. Grazia Cotticelli, Elisabetta Soragni, Robert B. Wilson, Marek Napierala, Jill S. Napierala. Molecular Therapy Methods & Clinical Development, Volume 32, Issue 4, 101347 DOI: 10.1016/j.omtm.2024.101347
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