| Size | Price | Stock | Qty |
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| 50mg |
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| Targets |
RTC13 targets premature termination codons (PTCs), also known as nonsense mutations, which result in truncated, non-functional proteins. It is a PTC readthrough inducer that promotes the incorporation of a near-cognate amino acid at the PTC, allowing translation to continue and producing full-length, functional protein. By restoring dystrophin expression in the mdx mouse model of Duchenne muscular dystrophy, RTC13 demonstrates its mechanism of action.
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| ln Vitro |
In vitro, RTC13 is a PTC readthrough inducer that promotes readthrough of premature termination codons. It restores dystrophin expression in cells carrying nonsense mutations in the dystrophin gene. These in vitro properties make RTC13 a valuable tool for studying nonsense mutation suppression and its potential for treating genetic disorders caused by PTCs.
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| ln Vivo |
In vivo, RTC13 restores dystrophin expression and improves muscle function in the mdx mouse model of Duchenne muscular dystrophy. These findings highlight its potential as a disease-modifying therapy targeting nonsense mutations in DMD and possibly other genetic disorders caused by PTCs. RTC13 offers a targeted, mutation-specific therapeutic approach for patients with nonsense mutations.
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| Enzyme Assay |
The in vitro enzyme/receptor binding assay for RTC13 is not applicable, as the compound is a PTC readthrough inducer that acts on the translation machinery rather than a specific enzyme or receptor. Its activity is assessed in cell-based assays measuring readthrough of a reporter gene containing a premature termination codon or by measuring restoration of full-length protein expression in cells with nonsense mutations.
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| Cell Assay |
The in vitro cell-based assay for RTC13 involves culturing cells carrying a reporter gene with a premature termination codon or cells with nonsense mutations in a gene of interest (e.g., dystrophin). Cells are treated with RTC13 at various concentrations, and readthrough activity is assessed by measuring reporter gene expression (e.g., luciferase activity) or by Western blot for restoration of full-length protein expression. Cell viability is assessed using MTT or CellTiter-Glo assays.
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| Animal Protocol |
In vivo animal studies for RTC13 are conducted in the mdx mouse model of Duchenne muscular dystrophy. Mice are administered RTC13 orally or intraperitoneally at various doses. Dystrophin expression is assessed by Western blot and immunohistochemistry in muscle tissue. Muscle function is assessed using grip strength, treadmill performance, or other functional tests. Standard protocols for the mdx mouse model are employed.
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| ADME/Pharmacokinetics |
The pharmacokinetic properties of RTC13 have been characterized in preclinical studies. As a small molecule with a molecular weight of 315.30 and a molecular formula of C14H9N3O4S, it is expected to have moderate oral bioavailability. The compound can be formulated for in vivo administration. Detailed PK parameters such as half-life, Cmax, and bioavailability are available from preclinical studies.
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| Toxicity/Toxicokinetics |
The toxicity profile of RTC13 has been evaluated in preclinical studies. As a PTC readthrough inducer, its primary safety concerns would relate to off-target effects on normal termination codons or effects on cellular translation. Standard toxicology assessments would include acute and sub-chronic toxicity studies in rodents, with endpoints including clinical signs, body weight, clinical pathology, and histopathology. No significant toxicity has been reported in available literature.
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| References |
1: Gómez-Grau M, Garrido E, Cozar M, Rodriguez-Sureda V, Domínguez C, Arenas C, Gatti RA, Cormand B, Grinberg D, Vilageliu L. Evaluation of Aminoglycoside and Non-Aminoglycoside Compounds for Stop-Codon Readthrough Therapy in Four Lysosomal Storage Diseases. PLoS One. 2015 Aug 19;10(8):e0135873. doi: 10.1371/journal.pone.0135873. eCollection 2015. PubMed PMID: 26287674; PubMed Central PMCID: PMC4545610. 2: Du L, Jung ME, Damoiseaux R, Completo G, Fike F, Ku JM, Nahas S, Piao C, Hu H, Gatti RA. A new series of small molecular weight compounds induce read through of all three types of nonsense mutations in the ATM gene. Mol Ther. 2013 Sep;21(9):1653-60. doi: 10.1038/mt.2013.150. Epub 2013 Jun 18. PubMed PMID: 23774824; PubMed Central PMCID: PMC3776636. 3: Kayali R, Ku JM, Khitrov G, Jung ME, Prikhodko O, Bertoni C. 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. doi: 10.1093/hmg/dds223. Epub 2012 Jun 12. PubMed PMID: 22692682; PubMed Central PMCID: PMC3607466. 4: Nakamura K, Du L, Tunuguntla R, Fike F, Cavalieri S, Morio T, Mizutani S, Brusco A, Gatti RA. Functional characterization and targeted correction of ATM mutations identified in Japanese patients with ataxia-telangiectasia. Hum Mutat. 2012 Jan;33(1):198-208. doi: 10.1002/humu.21632. Epub 2011 Nov 9. PubMed PMID: 22006793; PubMed Central PMCID: PMC3261637. 5: Jung ME, Ku JM, Du L, Hu H, Gatti RA. Synthesis and evaluation of compounds that induce readthrough of premature termination codons. Bioorg Med Chem Lett. 2011 Oct 1;21(19):5842-8. doi: 10.1016/j.bmcl.2011.07.107. Epub 2011 Aug 4. PubMed PMID: 21873052.
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| Additional Infomation |
RTC13 is a research compound and has not been approved for clinical use. It is a PTC readthrough inducer that restores dystrophin expression and improves muscle function in the mdx mouse model of Duchenne muscular dystrophy. RTC13 holds therapeutic potential for treating DMD patients with nonsense mutations and may be applicable to other genetic diseases caused by PTCs. It offers a targeted, mutation-specific therapeutic approach.
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| Molecular Formula |
C14H9N3O4S
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| Molecular Weight |
315.30
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| Exact Mass |
315.03
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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 # |
313530-30-2
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| Related CAS # |
1359825-94-7; 313530-30-2;
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| PubChem CID |
2865639
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| Appearance |
Solid powder
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| LogP |
2.5
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| Hydrogen Bond Donor Count |
1
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| Hydrogen Bond Acceptor Count |
5
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| Rotatable Bond Count |
2
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| Heavy Atom Count |
22
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| Complexity |
543
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| Defined Atom Stereocenter Count |
0
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| InChi Key |
CZZAPCPWFCGOCC-GHXNOFRVSA-N
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| InChi Code |
InChI=1S/C14H9N3O4S/c15-14-16-13(18)12(22-14)7-8-5-6-11(21-8)9-3-1-2-4-10(9)17(19)20/h1-7H,(H2,15,16,18)/b12-7-
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| Chemical Name |
(Z)-2-Imino-5-((5-(2-nitrophenyl)furan-2-yl)methylene)thiazolidin-4-one
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| Synonyms |
RTC13; RTC-13; RTC 13; Read-through compound 13; WAY301503; WAY-301503;; WAY 301503;
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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) |
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
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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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