CB096 selectively inhibited repeat associated non-ATG (RAN) translation and decreased the production of poly(GP) DPRs without affecting r(G4C2)66 mRNA levels in an HEK293T cellular model of c9ALS/FTD, alleviated dysfunctional nucleocytoplasmic transport, and selectively reduced the abundance of r(G4C2)66-induced stress granules. Moreover, CB096 is a selective binder to the r(G4C2)8 hairpin over r(G2C4)8 (antisense), r(GGCC)10 (base pair) RNAs, and other 1×1 internal loops.

KEYWORDS: CB096 | Supplier | RAN inhibitor | CB 096 | CB-096 | CAS [108883-90-5] | DNA-RNA | RAN | Inhibitor | Hypertension | Amyotrophic lateral sclerosis | ALS | Frontotemporal dementia | FTD