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Homocarbonyltopsentin

Cat No.:V32313 Purity: ≥98%
Homocarbonyltopsentin (PK4C9) is a small molecule compound that binds TSL2 and is a high carbonyl-forming protein.
Homocarbonyltopsentin
Homocarbonyltopsentin Chemical Structure CAS No.: 172286-77-0
Product category: New2
This product is for research use only, not for human use. We do not sell to patients.
Size Price Stock Qty
5mg
Other Sizes
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Product Description
Homocarbonyltopsentin (PK4C9) is a small molecule compound that binds TSL2 and is a high carbonyl-forming protein. It binds to the pentaloop conformation of TSL2 and promotes the transition to the clover conformation, enhancing SMN2 exon 7 (E7) splicing with EC50 of 16 μM.
Biological Activity I Assay Protocols (From Reference)
ln Vitro
When homocarbonyltopsentin (PK4C9) (10–40 μM) was applied for 24 hours, GM03813C cells expressed SMN2 isoforms, except for E7, up to 5.2 times less frequently than E7, and up to 3 times more frequently than E7 [1]. Homocarbonyltopsentin (PK4C9) (40 μM; 24 hours) increased SMN protein expression 1.5 times over GM03813C cells treated with DMSO [1].
Cell Assay
RT-PCR[1]
Cell Types: GM03813C (SMA) cells; GM03814B Fibroblast
Tested Concentrations: 10-40 μM
Incubation Duration: 24 hrs (hours)
Experimental Results: E7 inclusion rate reached a maximum of 91% (DMSO-treated cells increased by 25%).

Western Blot Analysis[1]
Cell Types: GM03813C (SMA) cells; GM03814B fibroblast
Tested Concentrations: 40 μM
Incubation Duration: 24 hrs (hours)
Experimental Results: SMN protein increased in SMA cells.
References
[1]. Garcia-Lopez A, et al. Targeting RNA structure in SMN2 reverses spinal muscular atrophy molecular phenotypes. Nat Commun. 2018 May 23;9(1):2032.
These protocols are for reference only. InvivoChem does not independently validate these methods.
Physicochemical Properties
Molecular Formula
C21H14N4O3
Molecular Weight
370.36086
Exact Mass
370.106
CAS #
172286-77-0
PubChem CID
11326167
Appearance
Typically exists as solid at room temperature
LogP
3.3
Hydrogen Bond Donor Count
4
Hydrogen Bond Acceptor Count
4
Rotatable Bond Count
4
Heavy Atom Count
28
Complexity
629
Defined Atom Stereocenter Count
0
InChi Key
DRCVQVIMGSWRLN-UHFFFAOYSA-N
InChi Code
InChI=1S/C21H14N4O3/c26-11-5-6-13-15(9-23-17(13)7-11)20(28)21-24-10-18(25-21)19(27)14-8-22-16-4-2-1-3-12(14)16/h1-10,22-23,26H,(H,24,25)
Chemical Name
[2-(6-hydroxy-1H-indole-3-carbonyl)-1H-imidazol-5-yl]-(1H-indol-3-yl)methanone
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)
DMSO : ~125 mg/mL (~337.51 mM)
Solubility (In Vivo)
Solubility in Formulation 1: ≥ 2.08 mg/mL (5.62 mM) (saturation unknown) in 10% DMSO + 40% PEG300 +5% Tween-80 + 45% Saline (add these co-solvents sequentially from left to right, and one by one), clear solution.
For example, if 1 mL of working solution is to be prepared, you can add 100 μL of 20.8 mg/mL clear DMSO stock solution to 400 μL PEG300 and mix evenly; then add 50 μL Tween-80 + to the above solution and mix evenly; then add 450 μL normal saline to adjust the volume to 1 mL.
Preparation of saline: Dissolve 0.9 g of sodium chloride in 100 mL ddH₂ O to obtain a clear solution.

 (Please use freshly prepared in vivo formulations for optimal results.)
Preparing Stock Solutions 1 mg 5 mg 10 mg
1 mM 2.7001 mL 13.5004 mL 27.0008 mL
5 mM 0.5400 mL 2.7001 mL 5.4002 mL
10 mM 0.2700 mL 1.3500 mL 2.7001 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

Molarity Calculator allows you to calculate the mass, volume, and/or concentration required for a solution, as detailed below:

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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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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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