Di-Ellipticine-RIBOTAC

Di-Ellipticine-RIBOTAC (Compound 7) is a RNA RIBOTAC degrader, capable of specifically binding and degrading r (G4C2) exp. Di-Ellipticine-RIBOTAC 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 significantly improves the pathological phenotype of c9ALS/FTD in cells and mouse models. Di-Ellipticine-RIBOTAC can be used for the study of c9ALS/FTD[1].

Product Specifications

CAS Number

[2767983-76-4]

UNSPSC

12352005

Target

PROTACs

Type

Reference compound

Related Pathways

PROTAC

Applications

Cancer-programmed cell death

Field of Research

Neurological Disease

Assay Protocol

https://www.medchemexpress.com/di-ellipticine-ribotac.html

Solubility

10 mM in DMSO

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

Molecular Formula

C78H87N7O16S

Molecular Weight

1410.63

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.