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].

Product Specifications

CAS Number

[2767983-77-5]

UNSPSC

12352005

Target

PROTACs

Related Pathways

PROTAC

Field of Research

Neurological Disease

Smiles

CCOC(C1=C(S/C(C1=O)=C\C2=CC=C(C(O)=C2)OCCOCCOCCOCCNC(CCC(N(CCOCCOCCOC3=CC4=C(NC5=C4C(C)=C6C=NC=CC6=C5C)C=C3)CCOCCOCCOC7=CC=C8NC9=C(C(C)=C%10C=NC=CC%10=C9C)C8=C7)=O)=O)NC%11=CC=CC=C%11)=O.OC(C(F)(F)F)=O

Molecular Formula

C80H88F3N7O18S

Molecular Weight

1524.65

References & Citations

[1]Bush JA, et al. Ribonuclease recruitment using a small molecule reduced c9ALS/FTD r (G4C2) repeat expansion in vitro and in vivo ALS models. Sci Transl Med. 2021 Oct 27;13 (617) :eabd5991.