Ataluren
Based on 8 publication(s) in Google Scholar
Ataluren (PTC124) is an orally available CFTR-G542X nonsense allele inhibitor.
For research use only. We do not sell to patients.
- Purity: 99.41%
- CAS No.: 775304-57-9
- Formula: C15H9FN2O3
- Molecular Weight:284.24
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Storage:Powder -20°C, 3 years , 4°C, 2 years ; In solvent -80°C, 2 years , -20°C, 1 year
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Biological Activity
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Chemical Information
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Solvent & Solubility
- Protocol
- Purity & Documentation
- References
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Help & FAQs
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Publications Citing Use of MedChemExpress (MCE) Ataluren
More- Mol Ther Nucleic Acids. 2023 Jun 26:33:227-239. [Abstract]
- Int J Mol Sci. 2020 Dec 31;22(1):344. [Abstract]
- Biomedicines. 2023 Apr 28;11(5):1310. [Abstract]
- Biomedicines. 2022 Nov 16;10(11):2948. [Abstract]
- Hum Mol Genet. 2020 Mar 13;29(4):624-634. [Abstract]
- Biopolymers. 2014 Apr;101(4):391-7. [Abstract]
- J Neurooncol. 2025 Mar;172(1):163-173. [Abstract]
- Patent. US20200199571A1
Customer Validation & Images
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IF
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IF
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RT-PCR
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Cell Proliferation/Viability Assay
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WB
Biological Activity
CFTR[1]
This premature "stop" signal (a class I mutation) prevents the cell from producing a full-length CFTR protein[1]. Ataluren (PTC124)-a new chemical entity that selectively induces ribosomal readthrough of premature but not normal termination codons[2].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
| NCT Number | Sponsor | Condition | Start Date |
Phase
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|---|---|---|---|---|
| NCT01329991 | Plexxikon| | 2011-05 | PHASE1 |
Chemical Information
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CAS No. 775304-57-9
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Appearance Solid
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Molecular Weight 284.24
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Formula C15H9FN2O3
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Color White to off-white
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SMILES
O=C(C1=CC=CC(C2=NOC(C3=CC=CC=C3F)=N2)=C1)O
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Synonyms
PTC124
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Shipping
Room temperature in continental US; may vary elsewhere.
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Storage
Powder -20°C 3 years 4°C 2 years In solvent -80°C 2 years -20°C 1 year
Publications (8)
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Journal Impact Factor
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Most Recent
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Mol Ther Nucleic Acids
Combining nonsense mutation suppression therapy with nonsense-mediated decay inhibition in neurofibromatosis type 1. [Abstract]2023 Jun 26:33:227-239. PMID: 37520682 -
Int J Mol Sci
Synergy between Readthrough and Nonsense Mediated Decay Inhibition in a Murine Model of Cystic Fibrosis Nonsense Mutations. [Abstract]2020 Dec 31;22(1):344. PMID: 33396210 -
Biomedicines
A Simple and Affordable Method to Create Nonsense Mutation Clones of p53 for Studying the Premature Termination Codon Readthrough Activity of PTC124. [Abstract]2023 Apr 28;11(5):1310. PMID: 37238980
Ataluren purchased from MedChemExpress. Usage Cited in: Biomedicines. 2023 Apr 28;11(5):1310. [Abstract]
The expression level of p53 was assessed using the p53 N-terminal antibody (DO-1) following treatment with either DMSO or Ataluren (PTC124) in four different p53 nonsense mutation clones. It was observed that Ataluren (PTC124) (50 μM; 48 h) was able to induce re-expression of p53 as red color in H1299-R306X and H1299-R342X clones but not in H1299-W91X and H1299-S94X clones. Hoechst 33342 was used to label the nucleus as blue color.
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Biomedicines
PTC124 Rescues Nonsense Mutation of Two Tumor Suppressor Genes NOTCH1 and FAT1 to Repress HNSCC Cell Proliferation. [Abstract]2022 Nov 16;10(11):2948. PMID: 36428516
Ataluren purchased from MedChemExpress. Usage Cited in: Biomedicines. 2022 Nov 16;10(11):2948. [Abstract]
Ataluren (PTC124) (50 μM; 48 h) could induce p53 expression in SAS cells.
Ataluren purchased from MedChemExpress. Usage Cited in: Biomedicines. 2022 Nov 16;10(11):2948. [Abstract]
Ataluren (PTC124) (50 μM; 48 h) could induce DDIT4 and repress CFGF expression in HO-1-u-1 cells (** p < 0.01, *** p < 0.001). DMSO only was calculated as 1 to normalize for the other conditions.
Ataluren purchased from MedChemExpress. Usage Cited in: Biomedicines. 2022 Nov 16;10(11):2948. [Abstract]
Ataluren (PTC124) (50 μM; 48 h) could repress HO-1-u-1 cell growth (* p < 0.05). Ataluren (PTC124) (50 μM; 48 h) could reverse TT-10 (10 μM)-mediated HO-1-u-1 cell proliferation (** p < 0.01). DMSO/MOCK was calculated as 1 to normalize for the other conditions.
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Hum Mol Genet
2020 Mar 13;29(4):624-634. PMID: 31913476 -
Biopolymers
Sequence context and crosslinking mechanism affect the efficiency of in vivo capture of a protein-protein interaction. [Abstract]2014 Apr;101(4):391-7. PMID: 24037947
Ataluren purchased from MedChemExpress. Usage Cited in: Biopolymers. 2014 Apr;101(4):391-7. [Abstract]
Western blot (α-FLAG) comparison of LexA+Gal4 Phe849pBpa expressed in the presence of increasing concentrations of Ataluren (PTC124). Additional details can be found in the Methods section.
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J Neurooncol
Single-cell RNA-seq reveals diverse molecular signatures associated with Methotrexate resistance in primary central nervous system lymphoma cells. [Abstract]2025 Mar;172(1):163-173. PMID: 39636551 -
Solvent & Solubility
DMSO : ≥ 52 mg/mL (182.94 mM; Hygroscopic DMSO has a significant impact on the solubility of product, please use newly opened DMSO)
* "≥" means soluble, but saturation unknown.
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, 2 years; -20°C, 1 year. When stored at -80°C, please use it within 2 years. When stored at -20°C, please use it within 1 year.
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, 2 years; -20°C, 1 year. When stored at -80°C, please use it within 2 years. When stored at -20°C, please use it within 1 year.
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
Select the appropriate dissolution method based on your experimental animal and administration route.
- For the following dissolution methods, please ensure to first prepare a clear stock solution using an In Vitro approach and then sequentially add co-solvents:
- To ensure reliable experimental results, the clarified stock solution can be appropriately stored based on storage conditions. As for the working solution for In Vivo experiments, it is recommended to prepare freshly and use it on the same day.
- The percentages shown for the solvents indicate their volumetric ratio in the final prepared solution. If precipitation or phase separation occurs during preparation, heat and/or sonication can be used to aid dissolution.
Add each solvent one by one: 10% DMSO 90% (20% SBE-β-CD in Saline)
Solubility: 2.5 mg/mL (8.80 mM); Suspended solution; Need ultrasonic
This protocol yields a suspended solution of 2.5 mg/mL. Suspended solution can be used for oral and intraperitoneal injection.
Taking 1 mL working solution as an example, add 100 μL DMSO stock solution (25.0 mg/mL) to 900 μL 20% SBE-β-CD in Saline, and mix evenly.
Preparation of 20% SBE-β-CD in Saline (4°C, storage for one week): 2 g SBE-β-CD powder is dissolved in 10 mL Saline, completely dissolve until clear.
Please enter the basic information of animal experiments:
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Recommended: Prepare an additional quantity of animals to account for potential losses during experiments.
Please enter your animal formula composition:
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%DMSO +
Recommended: Keep the proportion of DMSO in working solution below 2% if your animal is weak.
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%+
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+%Tween-80 + +
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%Saline +
The co-solvents required include: DMSO, . All of co-solvents are available by MedChemExpress (MCE). , Tween 80. All of co-solvents are available by MedChemExpress (MCE).
Working solution concentration: 0.22 mg/mL
Method for preparing stock solution: mg drug dissolved in μL DMSO. Stock solution concentration: mg/mL.
1. Take μL DMSO stock solution;
2. Add μL .
μL , mix evenly;
3. Then add μL Tween 80, mix evenly;
4. Then add μL
Please ensure that the stock solution in the first step is dissolved to a clear state, and add co-solvents in sequence. You can use ultrasonic heating (ultrasonic cleaner, recommended frequency 20-40 kHz), vortexing, etc. to assist dissolution.
Protocol
Duplicate samples of HEK293 cells harbouring LUC-190 (UGA) are incubated in the presence of 5 μM Ataluren (PTC124) (treated) or 1% DMSO (untreated) for 20 h. The cells are collected, washed twice in phosphate buffered saline (PBS), resuspended in sample buffer (Bio-Rad) and shipped on dry ice to Kendrick Laboratories for two-dimensional electrophoretic analysis Isoelectric focusing (pH 3.5-10) is carried out in glass tubes for 20,000 V-hours. One μg of a tropomyosin internal standard is added to each sample. Second dimension SDS slab gel electrophoresis is carried out for approximately 6 h at 25 mA per gel. After electrophoresis, gels are transferred to PVDF paper. Computerized analysis of spot mobility used Phoretix software[2].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
Mice[3]
Male mice are randomly assigned to either a treatment group or vehicle control group. Six to eight mice per group are treated with 10 or 100 mg/kg Ataluren (PTC124) dissolved in PBS containing DMSO (2% for 10 mg/kg and 20% for 100 mg/kg) and (2-hydroxypropyl)-β-cyclodextrin (22%) via intraperitoneal (i.p.) injections four times daily for 2 consecutive days. Six to eight control mice are treated with the vehicle of the drug: PBS containing DMSO (2% or 20%) and (2-hydroxypropyl)-β-cyclodextrin (22%). Immediately following the last injection on the second day, tissues are collected and stored at −80°C for further use.
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
Purity & Documentation
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Data Sheet (280 KB)
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SDS (396 KB)
- English - EN (396 KB)
- Français - FR (396 KB)
- Deutsch - DE (396 KB)
- Norwegian - NO (396 KB)
- Español - ES (396 KB)
- Swedish - SV (396 KB)
- Italian - IT (396 KB)
- Portuguese - PT (396 KB)
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Handling Instructions (2659 KB)
References
[1]. Pettit RS, et al. CFTR Modulators for the Treatment of Cystic Fibrosis. P T. 2014 Jul;39(7):500-11. [Content Brief]
[2]. Welch EM, et al. PTC124 targets genetic disorders caused by nonsense mutations. Nature, 2007, 447(7140), 87-91. [Content Brief]
[3]. Miller JN, et al. The novel Cln1(R151X) mouse model of infantile neuronal ceroid lipofuscinosis (INCL) for testing nonsense suppression therapy. Hum Mol Genet. 2015 Jan 1;24(1):185-96. [Content Brief]
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, 2 years; -20°C, 1 year. When stored at -80°C, please use it within 2 years. When stored at -20°C, please use it within 1 year.
| Optional Solvent | Concentration Solvent Mass | 1 mg | 5 mg | 10 mg | 25 mg |
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| DMSO | 1 mM | 3.5182 mL | 17.5908 mL | 35.1815 mL | 87.9538 mL |
| 5 mM | 0.7036 mL | 3.5182 mL | 7.0363 mL | 17.5908 mL | |
| 10 mM | 0.3518 mL | 1.7591 mL | 3.5182 mL | 8.7954 mL | |
| 15 mM | 0.2345 mL | 1.1727 mL | 2.3454 mL | 5.8636 mL | |
| 20 mM | 0.1759 mL | 0.8795 mL | 1.7591 mL | 4.3977 mL | |
| 25 mM | 0.1407 mL | 0.7036 mL | 1.4073 mL | 3.5182 mL | |
| 30 mM | 0.1173 mL | 0.5864 mL | 1.1727 mL | 2.9318 mL | |
| 40 mM | 0.0880 mL | 0.4398 mL | 0.8795 mL | 2.1988 mL | |
| 50 mM | 0.0704 mL | 0.3518 mL | 0.7036 mL | 1.7591 mL | |
| 60 mM | 0.0586 mL | 0.2932 mL | 0.5864 mL | 1.4659 mL | |
| 80 mM | 0.0440 mL | 0.2199 mL | 0.4398 mL | 1.0994 mL | |
| 100 mM | 0.0352 mL | 0.1759 mL | 0.3518 mL | 0.8795 mL |
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