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).
For research use only. We do not sell to patients.
- CAS No.: 2767983-76-4
- Formula: C78H87N7O16S
- Molecular Weight:1410.63
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Storage:
Please store the product under the recommended conditions in the Certificate of Analysis.
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Biological Activity
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Chemical Information
- Purity & Documentation
- References
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Help & FAQs
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Biological Activity
di-Ellipticine-RIBOTAC (compound 7) (5-500 nM) inhibits RAN translation of r(G4C2)66 in HEK293T cells, and reduces quantities of the r(G4C2)exp-containing transcript in a dose-dependent fashion[1].
di-Ellipticine-RIBOTAC (5-500 nM) selectively reduces C9orf72 intron 1 abundance with an IC50 of ~50 nM. di-Ellipticine-RIBOTAC also reduces poly(GP) abundance in c9ALS patient-derived lymphoblastoid cell lines (LCLs) and c9ALS patient-derived induced pluripotent stem cells (iPSCs)[1].
Di-Ellipticine-RIBOTAC (50 nM; 2.5 weeks) reduces C9orf72 transcripts containing the r(G4C2)exp repeat in intron 1 in iPSC-derived spinal motor neurons (iPSNs) from c9ALS patients and reversed the loss of Nup98[1].
di-Ellipticine-RIBOTAC cleaves Cy5- and Cy3-labeled r(G4C2)8 RNA in the presence of RNase L[1].
di-Ellipticine-RIBOTAC inhibits hnRNP H1 binding to r(G4C2)8 RNA (IC50 = 2.4 μM)[1].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
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Animal Model:+/+PWR500 (12-25 weeks old) (expressing 500 r(G4C2) repeats)[1]
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Dosage:33 nmol
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Administration:i.c.v.; single dose
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Result:Reduced r(G4C2)exp-containing intron 1 mRNA abundance by 44% compared to vehicle-treated controls, decreased the number of r(G4C2)exp-containing nuclear foci, reduced poly(GP) abundance by 74% in brain tissue, decreased poly(GP) and poly(GA) aggregates, and decreased TDP-43 inclusions.
Chemical Information
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CAS No. 2767983-76-4
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Molecular Weight 1410.63
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Formula C78H87N7O16S
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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
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Shipping
Room temperature in continental US; may vary elsewhere.
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Storage
Please store the product under the recommended conditions in the Certificate of Analysis.
Purity & Documentation
References
Calculators
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
di-Ellipticine-RIBOTAC Related Classifications
HY-141878 Related Classifications
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- di-Ellipticine-RIBOTAC
- 2767983-76-4
- RIBOTAC
- DNA/RNA Synthesis
- RNase L
- C9orf72
- rodent model
- r(G2C4)exp
- c9ALS/FTD
- FVB/NJ-Tg(C9orf72)500Lpwr/J (PWR500) mice
- HEK293T cells
- dipeptide repeat proteins
- Neurological Disease
- c9ALS patient-derived iPSCs
- c9ALS patient-derived iPSNs
- c9ALS patient-derived spinal neurons
- Nup98
- hnRNP H1
- c9ALS patient-derived LCLs
- Inhibitor
- inhibitor
- inhibit