di-Ellipticine-RIBOTAC TFA

di-Ellipticine-RIBOTAC TFA is a RNase recruiting chimera (RIBOTAC) degrader, capable of specifically binding and degrading expanded G4C2 RNA repeat (r(G₄C₂)^exp). di-Ellipticine-RIBOTAC TFA selectively binds the three-dimensional (3D) structure formed by r(G₄C₂)^exp and that recruits an endogenous ribonuclease (RNase) to cleave r(G₄C₂)^exp. di-Ellipticine-RIBOTAC TFA selectively degrades the mutant chromosome 9 open reading frame 72 (C9orf72) allele and reduces quantities of toxic dipeptide repeat proteins (DPRs) translated from r(G₄C₂)^exp. di-Ellipticine-RIBOTAC TFA significantly improves the pathological phenotype of amyotrophic lateral sclerosis/ frontotemporal dementia (c9ALS/FTD) in cells and mouse models. di-Ellipticine-RIBOTAC TFA can be used for the study of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

• Data Sheet (271 KB)