A GGGGCC repeat expansion in C9orf72 is the most common genetic cause of ALS and FTD (C9ALS/FTD). Dipeptide repeat (DPR) proteins, generated by translation of the expanded repeat, are a major pathogenic feature of C9ALS/FTD pathology, but their physiological impact has yet to be fully determined. He...
ORGANISM(S): Mus musculus (Mouse)