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) [1].

C22H20N10S2

488.59

NC(N/N=C/C1=CC=CC(C2=CC=C(S2)C3=CC=C(S3)C4=CC=CC(/C=N/NC(N)=N)=N4)=N1)=N

DNA/RNA Synthesis

Room temperature