RNA binder 1

RNA binder 1 (Compound 4b) is an RNA-binding agent capable of crossing the blood-brain barrier. It selectively binds to the G-quadruplex structure of the G4C2 repeat sequence RNA of the C9orf72 gene. This compound significantly reduces levels of the toxic polypeptides poly (GA) and poly (GP) in cells derived from amyotrophic lateral sclerosis (ALS) patients, while it has no significant impact on the antisense polypeptide poly (PR), demonstrating selectivity for sense RNA. RNA binder 1 is useful for studying ALS and frontotemporal dementia (FTD) .