2767983-77-5
Chemical Structure
di-Ellipticine-RIBOTAC TFA
- CAS No.: 2767983-77-5
- Formula:C80H88F3N7O18S
- Molecular Weight:1524.65
IUPAC Name: 2,2,2-trifluoroacetic acid compound with 4-(3-((1-(4-chlorophenyl)-2-oxo-2-(6-(trifluoromethoxy)indolin-1-yl)ethyl)amino)-5-methoxyphenoxy)butanoic acid (1:1)
InChIKey: MQRNDCLSCUUTIW-ZYURSNJDSA-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.OC(C(F)(F)F)=O
Biological Activity: 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)[1].
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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). | |||||||||||||||||||||
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Keywords
