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RuvBL1/2 reduce toxic dipeptide repeat protein burden in multiple models of C9orf72-ALS/FTD

Enhancing RuvBL1, but particularly RuvBL2 expression, reduces toxic dipeptide repeat proteins in vitro and in vivo models of C9orf72-linked ALS/FTD, suggesting that modulating RuvBL1/2 levels could be a promising therapeutic approach for C9ALS/FTD. A G4C2 hexanucleotide repeat expansion in C9orf72 i...

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Autori principali: Christopher P Webster, Bradley Hall, Olivia M Crossley, Dana Dauletalina, Marianne King, Ya-Hui Lin, Lydia M Castelli, Zih-Liang Yang, Ian Coldicott, Ergita Kyrgiou-Balli, Adrian Higginbottom, Laura Ferraiuolo, Kurt J De Vos, Guillaume M Hautbergue, Pamela J Shaw, Ryan JH West, Mimoun Azzouz
Natura: Artigo
Lingua:Inglês
Pubblicazione: Life Science Alliance LLC 2025-02-01
Serie:Life Science Alliance
Accesso online:https://www.life-science-alliance.org/lookup/doi/10.26508/lsa.202402757
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