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di-Ellipticine-RIBOTAC TFA

Dielliptic pyridine-RIBOTAC TFA is an RNase recruitment chimera (RIBOTAC) degrader that specifically binds to and degrades amplified G4C2 RNA repeat sequences (r(G4C2)exp).
di-Ellipticine-RIBOTAC TFA
di-Ellipticine-RIBOTAC TFA Chemical Structure CAS No.: 2767983-77-5
Product category: DNA(RNA) Synthesis
This product is for research use only, not for human use. We do not sell to patients.
Size Price
500mg
1g
Other Sizes

Other Forms of di-Ellipticine-RIBOTAC TFA:

  • di-Ellipticine-RIBOTAC
Official Supplier of:
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Top Publications Citing lnvivochem Products
Product Description
di-Ellipticine-RIBOTAC TFA is an RNase recruitment chimera (RIBOTAC) degrader that specifically binds to and degrades amplified G4C2 RNA repeat sequences (r(G4C2)exp). di-Ellipticine-RIBOTAC TFA selectively binds to the three-dimensional (3D) structure formed by r(G4C2)exp and recruits endogenous ribonucleases (RNases) to cleave r(G4C2)exp. di-Ellipticine-RIBOTAC TFA selectively degrades the mutant chromosome 9 open reading frame 72 (C9orf72) allele and reduces the amount of toxic dipeptide repeats (DPRs) produced by the translation of r(G4C2)exp. di-Ellipticine-RIBOTAC TFA significantly improves the pathological phenotype of amyotrophic lateral sclerosis/frontotemporal dementia (c9ALS/FTD) in cell and animal models. di-Ellipticine-RIBOTAC TFA can be used in research on amyotrophic lateral sclerosis (ALS) and frontotemporal dementia.
Biological Activity I Assay Protocols (From Reference)
ln Vitro
di-Ellipticine-RIBOTAC (compound 7) (5-500 nM) TFA inhibited RAN translation of r(G4C2)66 in HEK293T cells and reduced transcripts containing r(G4C2)exp in a dose-dependent manner [1]. di-Ellipticine-RIBOTAC (5-500 nM) TFA selectively reduced the abundance of C9orf72 intron 1 with an IC50 of approximately 50 nM. di-Ellipticine-RIBOTAC TFA also reduced the abundance of poly(GP) in lymphoblastic cell lines (LCLs) and induced pluripotent stem cells (iPSCs) derived from c9ALS patients [1]. di-Ellipticine-RIBOTAC (50 nM; 2.5 weeks) TFA reduced C9orf72 transcripts containing intron 1 of r(G4C2)exp in iPSC-derived spinal neurons (iPSNs) from c9ALS patients and reversed the loss of Nup98[1]. di-Ellipticine-RIBOTAC TFA cleaved Cy5- and Cy3-labeled r(G4C2)8 RNA in the presence of RNase L[1]. di-Ellipticine-RIBOTAC TFA inhibited the binding of hnRNP H1 to r(G4C2)8 RNA (IC50 = 2.4 μM)[1].
ln Vivo
di-Ellipticine-RIBOTAC (compound 7) (33 nmol; intraventricular injection; single solution) TFA attenuated c9ALS/FTD pathology in a C9orf72 cytokine cell model (+/+PWR500)[1]. +/+PWR500 (12-25 weeks old) (expressing 500 r(G4C2) repeats)[1] 33 nanomolar ICV; compared with the mediator-treated control, a single dose reduced the abundance of intron 1 mRNA containing r(G4C2)exp by 44%, reduced the number of nuclear foci containing r(G4C2)exp, reduced the abundance of aggregate (GP) in brain tissue by 74%, reduced aggregate (GP) and aggregate (GA) aggregates, and reduced TDP-43 inclusions.
References

[1]. https://pubmed.ncbi.nlm.nih.gov/34705518/

These protocols are for reference only. InvivoChem does not independently validate these methods.
Physicochemical Properties
Molecular Formula
C80H88F3N7O18S
Molecular Weight
1524.65
CAS #
2767983-77-5
Related CAS #
di-Ellipticine-RIBOTAC; 2767983-76-4
Appearance
Typically exists as solids at room temperature
HS Tariff Code
2934.99.9001
Storage

Powder      -20°C    3 years

                     4°C     2 years

In solvent   -80°C    6 months

                  -20°C    1 month

Shipping Condition
Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)
Solubility Data
Solubility (In Vitro)
May dissolve in DMSO (in most cases), if not, try other solvents such as H2O, Ethanol, or DMF with a minute amount of products to avoid loss of samples
Solubility (In Vivo)
Note: Listed below are some common formulations that may be used to formulate products with low water solubility (e.g. < 1 mg/mL), you may test these formulations using a minute amount of products to avoid loss of samples.

Injection Formulations
(e.g. IP/IV/IM/SC)
Injection Formulation 1: DMSO : Tween 80: Saline = 10 : 5 : 85 (i.e. 100 μL DMSO stock solution 50 μL Tween 80 850 μL Saline)
*Preparation of saline: Dissolve 0.9 g of sodium chloride in 100 mL ddH ₂ O to obtain a clear solution.
Injection Formulation 2: DMSO : PEG300Tween 80 : Saline = 10 : 40 : 5 : 45 (i.e. 100 μL DMSO 400 μLPEG300 50 μL Tween 80 450 μL Saline)
Injection Formulation 3: DMSO : Corn oil = 10 : 90 (i.e. 100 μL DMSO 900 μL Corn oil)
Example: Take the Injection Formulation 3 (DMSO : Corn oil = 10 : 90) as an example, if 1 mL of 2.5 mg/mL working solution is to be prepared, you can take 100 μL 25 mg/mL DMSO stock solution and add to 900 μL corn oil, mix well to obtain a clear or suspension solution (2.5 mg/mL, ready for use in animals).
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Injection Formulation 4: DMSO : 20% SBE-β-CD in saline = 10 : 90 [i.e. 100 μL DMSO 900 μL (20% SBE-β-CD in saline)]
*Preparation of 20% SBE-β-CD in Saline (4°C,1 week): Dissolve 2 g SBE-β-CD in 10 mL saline to obtain a clear solution.
Injection Formulation 5: 2-Hydroxypropyl-β-cyclodextrin : Saline = 50 : 50 (i.e. 500 μL 2-Hydroxypropyl-β-cyclodextrin 500 μL Saline)
Injection Formulation 6: DMSO : PEG300 : castor oil : Saline = 5 : 10 : 20 : 65 (i.e. 50 μL DMSO 100 μLPEG300 200 μL castor oil 650 μL Saline)
Injection Formulation 7: Ethanol : Cremophor : Saline = 10: 10 : 80 (i.e. 100 μL Ethanol 100 μL Cremophor 800 μL Saline)
Injection Formulation 8: Dissolve in Cremophor/Ethanol (50 : 50), then diluted by Saline
Injection Formulation 9: EtOH : Corn oil = 10 : 90 (i.e. 100 μL EtOH 900 μL Corn oil)
Injection Formulation 10: EtOH : PEG300Tween 80 : Saline = 10 : 40 : 5 : 45 (i.e. 100 μL EtOH 400 μLPEG300 50 μL Tween 80 450 μL Saline)


Oral Formulations
Oral Formulation 1: Suspend in 0.5% CMC Na (carboxymethylcellulose sodium)
Oral Formulation 2: Suspend in 0.5% Carboxymethyl cellulose
Example: Take the Oral Formulation 1 (Suspend in 0.5% CMC Na) as an example, if 100 mL of 2.5 mg/mL working solution is to be prepared, you can first prepare 0.5% CMC Na solution by measuring 0.5 g CMC Na and dissolve it in 100 mL ddH2O to obtain a clear solution; then add 250 mg of the product to 100 mL 0.5% CMC Na solution, to make the suspension solution (2.5 mg/mL, ready for use in animals).
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Oral Formulation 3: Dissolved in PEG400
Oral Formulation 4: Suspend in 0.2% Carboxymethyl cellulose
Oral Formulation 5: Dissolve in 0.25% Tween 80 and 0.5% Carboxymethyl cellulose
Oral Formulation 6: Mixing with food powders


Note: Please be aware that the above formulations are for reference only. InvivoChem strongly recommends customers to read literature methods/protocols carefully before determining which formulation you should use for in vivo studies, as different compounds have different solubility properties and have to be formulated differently.

 (Please use freshly prepared in vivo formulations for optimal results.)
Preparing Stock Solutions 1 mg 5 mg 10 mg
1 mM 0.6559 mL 3.2794 mL 6.5589 mL
5 mM 0.1312 mL 0.6559 mL 1.3118 mL
10 mM 0.0656 mL 0.3279 mL 0.6559 mL

*Note: Please select an appropriate solvent for the preparation of stock solution based on your experiment needs. For most products, DMSO can be used for preparing stock solutions (e.g. 5 mM, 10 mM, or 20 mM concentration); some products with high aqueous solubility may be dissolved in water directly. Solubility information is available at the above Solubility Data section. Once the stock solution is prepared, aliquot it to routine usage volumes and store at -20°C or -80°C. Avoid repeated freeze and thaw cycles.

Calculator

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An example of molarity calculation using the molarity calculator is shown below:
What is the mass of compound required to make a 10 mM stock solution in 5 ml of DMSO given that the molecular weight of the compound is 350.26 g/mol?
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Dilution Calculator allows you to calculate how to dilute a stock solution of known concentrations. For example, you may Enter C1, C2 & V2 to calculate V1, as detailed below:

What volume of a given 10 mM stock solution is required to make 25 ml of a 25 μM solution?
Using the equation C1V1 = C2V2, where C1=10 mM, C2=25 μM, V2=25 ml and V1 is the unknown:
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Note: Chemical formula is case sensitive: C12H18N3O4  c12h18n3o4
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Definitions of molecular mass, molecular weight, molar mass and molar weight:
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In vivo Formulation Calculator (Clear solution)
Step 1: Enter information below (Recommended: An additional animal to make allowance for loss during the experiment)
Step 2: Enter in vivo formulation (This is only a calculator, not the exact formulation for a specific product. Please contact us first if there is no in vivo formulation in the solubility section.)
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Calculation results

Working concentration mg/mL;

Method for preparing DMSO stock solution mg drug pre-dissolved in μL DMSO (stock solution concentration mg/mL). Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug.

Method for preparing in vivo formulation:Take μL DMSO stock solution, next add μL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O,mix and clarify.

(1) Please be sure that the solution is clear before the addition of next solvent. Dissolution methods like vortex, ultrasound or warming and heat may be used to aid dissolving.
             (2) Be sure to add the solvent(s) in order.

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