Article
VCP modulation ameliorates pathological features in C9orf72 models.
Cell death & disease - 17 May 2026
Ferrari Veronica, Tedesco Barbara, Cozzi Marta, Pramaggiore Paola, Gagliani Maria Cristina, Magdalena Rocio, Cornaggia Laura, Casarotto Elena, Chierichetti Marta, Mohamed Ali, Brodnanovà Maria, Milioto Carmelo, Piccolella Margherita, Galbiati Mariarita, Crippa Valeria, Provenzani Alessandro, Cortese Katia, Rusmini Paola, Cristofani Riccardo, Poletti Angelo
Abstract excerpt
Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) are devastating neurodegenerative diseases linked by similar pathological mechanisms, which, in some familial forms, may be associated with the same genetic alterations. Among them, the most common is the C9ORF72 (C9) mutation. The C9 mutation consists in an aberrant expansion of the hexanucleotide repeat (G4C2)n that leads to the production...
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