Di-Ellipticine-RIBOTAC (TFA)

Di-Ellipticine-RIBOTAC TFA (Compound 7) is a RNA RIBOTAC degrader, capable of specifically binding and degrading r (G4C2) exp. Di-Ellipticine-RIBOTAC TFA only cuts the expanded GGGGCC repeat RNA in the pathogenic factor of amyotrophic lateral sclerosis/ frontotemporal dementia (c9ALS/FTD) - the chromosome 9 open reading frame 72 (9orf72) gene (r (G4C2) exp), and has no effect on normal short repetitive sequences. Di-Ellipticine-RIBOTAC TFA significantly improves the pathological phenotype of c9ALS/FTD in cells and mouse models. Di-Ellipticine-RIBOTAC TFA can be used for the study of c9ALS/FTD[1].