RNA binder 1

RNA binder 1 (Compound 4b) is a blood-brain permeable RNA binder. RNA binder 1 can selectively bind to the G-quadruplex structure of the G4C2 repeat sequence RNA of the C9orf72 gene. RNA binder 1 significantly reduces the levels of toxic polypeptides poly(GA) and poly(GP) produced by the G4C2 repeat sequence in amyotrophic lateral sclerosis (ALS) patient-derived cells. RNA binder 1 has no significant effect on the antisense polypeptide poly(PR), showing selectivity for sense RNA. RNA binder 1 can be used in the study of ALS and frontotemporal dementia (FTD).

• Data Sheet (269 KB)