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Article

Metformin inhibits RAN translation through PKR pathway and mitigates disease in C9orf72 ALS/FTD mice

20 Jul 2020

Abstract excerpt

Significance Repeat-associated non-AUG (RAN) proteins accumulate in patient brains and contribute to a growing number of neurodegenerative diseases. There is an urgent need to understand why expression of these proteins does not require canonical or near-cognate AUG start codons and to develop ways to block RAN protein production. We show several types of repeat-expansion RNAs activate the double-stranded...

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PMID: 32690681
DOI: 10.1073/pnas.2005748117

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