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| Targets |
RTC13 targets the ribosome and promotes read-through of premature termination codons (PTCs). By suppressing the recognition of PTCs as stop signals, it allows the translation machinery to continue past the mutation, resulting in the production of full-length functional protein. This mechanism has therapeutic potential for genetic disorders caused by nonsense mutations, such as Duchenne muscular dystrophy.
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| ln Vitro |
RTC13 promotes ribosomal read-through activity of premature termination codons (PTCs) in vitro. It restores dystrophin expression in cell-based models of Duchenne muscular dystrophy. This demonstrates its potent activity as a read-through agent.
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| ln Vivo |
RTC13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy (DMD). This confirms its in vivo efficacy in a relevant animal model of a nonsense mutation disorder. It is a promising read-through agent for the treatment of genetic disorders caused by nonsense mutations.
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| Enzyme Assay |
The in vitro activity of RTC13 is assessed using cell-based read-through assays. Cells harboring a reporter gene with a premature termination codon (PTC) are treated with various concentrations of RTC13. The production of full-length reporter protein is measured, and the read-through efficiency is calculated.
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| Cell Assay |
Cell-based assays are used to evaluate RTC13's read-through activity. Cells expressing a mutated gene with a PTC (such as dystrophin in DMD models) are treated with RTC13. The restoration of full-length protein expression is assessed by Western blot or immunofluorescence. The functional consequences of protein restoration, such as improved cell survival or function, can also be assessed.
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| Animal Protocol |
Animal/Disease Models: 10 to 12 weeks old wild-type (C57BL/10) mice [1].
Doses: 10 (n = 5), 30 (n = 5), 60 (n = 5) or 300 mg/kg (n = 5). Mode of Route of Administration: intraperitonealadministration. Experimental Results: Liver and kidney weights were unchanged at autopsy, and hematoxylin and eosin (H&E) staining demonstrated no histopathological changes. RTC13 has been evaluated in the mdx mouse model of Duchenne muscular dystrophy. In these studies, the compound is typically administered to mdx mice, and its effects on dystrophin expression and muscle function are assessed. Muscle function is evaluated using tests such as grip strength or treadmill performance. |
| ADME/Pharmacokinetics |
RTC13 has a molecular weight of 315.3 and a molecular formula of C14H9N3O4S. It is soluble in DMSO (20 mg/mL). It appears as a yellow to orange solid. Powder should be stored at -20°C for up to 3 years. Purity is typically 98%. RTC13 is a cell-permeable thiazolidinone read-through agent.
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| Toxicity/Toxicokinetics |
RTC13 is a research compound and its full toxicological profile is not detailed in the available literature. In the mdx mouse model, it has been used to restore dystrophin expression and improve muscle function without significant adverse effects reported. Standard safety precautions should be followed when handling the compound.
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| References |
[1]. Refik Kayali, et al. Read-through compound 13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy. Hum Mol Genet. 2012 Sep 15;21(18):4007-20.
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| Additional Infomation |
RTC13 (CAS#: 1359825-94-7) is a cell-permeable thiazolidinone derivative that promotes ribosomal read-through of premature termination codons (PTCs). It restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy. RTC13 is a promising read-through agent for the treatment of genetic disorders caused by nonsense mutations and is a valuable research tool for studying PTC read-through mechanisms.
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| Molecular Formula |
C14H9N3O4S
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| Molecular Weight |
315.3
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| Exact Mass |
315.031
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| Elemental Analysis |
C, 53.33; H, 2.88; N, 13.33; O, 20.30; S, 10.17
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| CAS # |
1359825-94-7
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| Appearance |
Solid powder
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| LogP |
3.442
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| SMILES |
S1C(=N)NC(C1=CC1=CC=C(C2C=CC=CC=2[N+](=O)[O-])O1)=O
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| Synonyms |
RTC 13; RTC-13; RTC13
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| HS Tariff Code |
2934.99.03.00
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| 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)
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| Solubility (In Vitro) |
DMSO : ~100 mg/mL (~317.16 mM)
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| 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
Injection Formulation 1: DMSO : Tween 80： Saline = 10 : 5 : 85 (i.e. 100 μL DMSO stock solution → 50 μL Tween 80 → 850 μL Saline)(e.g. IP/IV/IM/SC) *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 : PEG300 ：Tween 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). View More
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)] 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). View More
Oral Formulation 3: Dissolved in PEG400 (Please use freshly prepared in vivo formulations for optimal results.) |
| Preparing Stock Solutions | 1 mg | 5 mg | 10 mg | |
| 1 mM | 3.1716 mL | 15.8579 mL | 31.7158 mL | |
| 5 mM | 0.6343 mL | 3.1716 mL | 6.3432 mL | |
| 10 mM | 0.3172 mL | 1.5858 mL | 3.1716 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.
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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