Pharmaco-genetic evidence of presymptomatic lysosomal dysfunction coupled to detrimental DPR secretion in Drosophila Models of C9orf72-linked ALS/FTD.
Hexanucleotide repeat expansions in C9orf72, the most common genetic cause of Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), drive