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Cell cycle dysregulation contributes to neurodegeneration in human neurons and defines a druggable vulnerability in <i>C9orf72</i> ALS/FTD

2025-06-08

Abstract excerpt

<h4>ABSTRACT</h4> The C9orf72 hexanucleotide repeat expansion GGGGCC (G4C2) cause the most common genetic forms of ALS and frontotemporal dementia, affecting thousands of patients worldwide with uniformly fatal outcomes. C9orf72 ALS/FTD patients lack targeted treatments because druggable molecular vulnerabilities remain unidentified. Using iPSC-derived motor neurons from C9orf72 carriers and age-matched contro...

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Literature Corpus work
03bedc81-0f53-520e-9042-a518c53bd430
DOI
10.1101/2025.06.04.657714
Open publication

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