2767983-76-4
Chemical Structure
di-Ellipticine-RIBOTAC
- CAS No.: 2767983-76-4
- Formula:C78H87N7O16S
- Molecular Weight:1410.63
IUPAC Name: ethyl (Z)-5-(4-((25-((5,11-dimethyl-6H-pyrido[4,3-b]carbazol-9-yl)oxy)-17-(2-(2-(2-((5,11-dimethyl-6H-pyrido[4,3-b]carbazol-9-yl)oxy)ethoxy)ethoxy)ethyl)-13,16-dioxo-3,6,9,20,23-pentaoxa-12,17-diazapentacosyl)oxy)-3-hydroxybenzylidene)-4-oxo-2-(phenylamino)-4,5-dihydrothiophene-3-carboxylate
InChIKey: OVTKAOITTCSMRM-WERQLJTPSA-N
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
Biological Activity: di-Ellipticine-RIBOTAC is a RNase recruiting chimera (RIBOTAC) degrader, capable of specifically binding and degrading expanded G4C2 RNA repeat (r(G4C2)exp). di-Ellipticine-RIBOTAC 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 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 significantly improves the pathological phenotype of amyotrophic lateral sclerosis/ frontotemporal dementia (c9ALS/FTD) in cells and mouse models. di-Ellipticine-RIBOTAC can be used for the study of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD)[1].
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di-Ellipticine-RIBOTAC | di-Ellipticine-RIBOTAC is a RNase recruiting chimera (RIBOTAC) degrader, capable of specifically binding and degrading expanded G4C2 RNA repeat (r(G4C2)exp). di-Ellipticine-RIBOTAC 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 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 significantly improves the pathological phenotype of amyotrophic lateral sclerosis/ frontotemporal dementia (c9ALS/FTD) in cells and mouse models. di-Ellipticine-RIBOTAC can be used for the study of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). | |||||||||||||||||||||
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