A multimodal screening platform for endogenous dipeptide repeat proteins in C9orf72 patient iPSC neurons

A multimodal screening platform for endogenous dipeptide repeat proteins in C9orf72 patient iPSC neurons

Summary: Repeat expansions in C9orf72 are the most common cause of amyotrophic lateral sclerosis and frontotemporal dementia. Repeat-associated non-AUG (RAN) translation generates neurotoxic dipeptide repeat proteins (DPRs). To study endogenous DPRs, we inserted the minimal HiBiT luciferase reporter...

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Main Authors: Benedikt V. Hölbling, Yashica Gupta, Paolo M. Marchi, Magda L. Atilano, Michael Flower, Enric Ureña, Rajkumar A. Goulden, Hannah K. Dobbs, Eszter Katona, Alla Mikheenko, Ashling Giblin, Ali Raza Awan, Chloe L. Fisher-Ward, Niamh O'Brien, Deniz Vaizoglu, Liam Kempthorne, Katherine M. Wilson, Lauren M. Gittings, Mireia Carcolé, Marc-David Ruepp, Sarah Mizielinska, Linda Partridge, Pietro Fratta, Sarah J. Tabrizi, Bhuvaneish T. Selvaraj, Siddharthan Chandran, Emma Armstrong, Paul Whiting, Adrian M. Isaacs
Format: Article
Language:English
Published: Elsevier 2025-05-01
Series:Cell Reports
Subjects:
CP: Neuroscience
Online Access:http://www.sciencedirect.com/science/article/pii/S2211124725004668
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