PTC258
Based on 1 Customer Validation
PTC258 is a specific and orally active splicing modulator of Elongator complex protein 1 gene (ELP1). PTC258 increases the expression of ELP1 in vitro and in vivo. PTC258 is well tolerated in mouse model.
For research use only. We do not sell to patients.
- Purity : 99.84%
- CAS No.: 2476724-74-8
- Formula: C16H18ClN3S2
- Molecular Weight:351.92
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Storage:
-20°C, protect from light, stored under nitrogen
* In solvent : -80°C, 6 months; -20°C, 1 month (protect from light, stored under nitrogen)
All DNA/RNA Synthesis Isoforms
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Biological Activity
Description
In Vitro
In Vivo
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
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Animal Model:Familial Dysautonomia (FD) Mouse model[1]
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Dosage:3 mg/kg, 6 mg/kg, 12 mg/kg, 24 mg/kg
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Administration:Oral gavage; once daily for 3 month
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Result:Increased full-length ELP1 transcript in a dose-dependent manner. And also, it increased in functional ELP1 protein in the brain, trigeminal, liver, and quadricep.
Chemical Information
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CAS No. 2476724-74-8
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Appearance Solid
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Molecular Weight 351.92
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Formula C16H18ClN3S2
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Color White to yellow
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SMILES
CC(C1=NC(Cl)=CC(NCC2=CC=CS2)=C1S3)=C3C[C@H](C)N
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Shipping
Room temperature in continental US; may vary elsewhere.
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Storage
-20°C, protect from light, stored under nitrogen
* In solvent : -80°C, 6 months; -20°C, 1 month (protect from light, stored under nitrogen)
Solvent & Solubility
In Vitro:
DMSO : 100 mg/mL (284.16 mM; Need ultrasonic; Hygroscopic DMSO has a significant impact on the solubility of product, please use newly opened DMSO)
Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (protect from light, stored under nitrogen). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.
Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (protect from light, stored under nitrogen). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
Protocols
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RNA extraction experimental
By lysing cells, releasing RNA, and removing impurities such as proteins and DNA, high-purity RNA products are finally obtained. The commonly used traditional method is the guanidine isothiocyanate/phenol/chloroform method (Trizol), which is suitable for a variety of animal materials including animal tissues, microorganisms, cultured cells, etc., and most plant materials.
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How to Choose the Right Model Animal
Choosing the right model animal is a validity-driven decision in which the species, strain, sex, age, genetic background, disease-induction method, outcome measures, and welfare burden must match the scientific question rather than laboratory tradition or convenience. A model should be selected by judging face validity, construct validity, and predictive validity: whether it resembles the human phenotype, whether it reproduces relevant mechanisms, and whether results are likely to predict human biology or treatment response. Animal studies often fail to translate because of species differences, weak disease resemblance, poor experimental design, inadequate reporting, publication bias, and underuse of randomization, blinding, and sample-size justification. Unresolved questions include how to rank competing models objectively, how much human-disease complexity must be reproduced for a given objective, and when non-animal systems such as organoids, ex vivo tissue, or computational models
Purity & Documentation
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Data Sheet (273 KB)
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SDS (252 KB)
- English - EN (252 KB)
- Français - FR (252 KB)
- Deutsch - DE (252 KB)
- Norwegian - NO (252 KB)
- Español - ES (252 KB)
- Swedish - SV (252 KB)
- Italian - IT (252 KB)
- Korean - KR (252 KB)
- Portuguese - PT (252 KB)
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Handling Instructions (2659 KB)
References
Complete Stock Solution Preparation Table
Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (protect from light, stored under nitrogen). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.
| Optional Solvent | Concentration Solvent Mass | 1 mg | 5 mg | 10 mg | 25 mg |
|---|---|---|---|---|---|
| DMSO | 1 mM | 2.8416 mL | 14.2078 mL | 28.4155 mL | 71.0389 mL |
| 5 mM | 0.5683 mL | 2.8416 mL | 5.6831 mL | 14.2078 mL | |
| 10 mM | 0.2842 mL | 1.4208 mL | 2.8416 mL | 7.1039 mL | |
| 15 mM | 0.1894 mL | 0.9472 mL | 1.8944 mL | 4.7359 mL | |
| 20 mM | 0.1421 mL | 0.7104 mL | 1.4208 mL | 3.5519 mL | |
| 25 mM | 0.1137 mL | 0.5683 mL | 1.1366 mL | 2.8416 mL | |
| 30 mM | 0.0947 mL | 0.4736 mL | 0.9472 mL | 2.3680 mL | |
| 40 mM | 0.0710 mL | 0.3552 mL | 0.7104 mL | 1.7760 mL | |
| 50 mM | 0.0568 mL | 0.2842 mL | 0.5683 mL | 1.4208 mL | |
| 60 mM | 0.0474 mL | 0.2368 mL | 0.4736 mL | 1.1840 mL | |
| 80 mM | 0.0355 mL | 0.1776 mL | 0.3552 mL | 0.8880 mL | |
| 100 mM | 0.0284 mL | 0.1421 mL | 0.2842 mL | 0.7104 mL |