RNA binder 1

Description

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 .