1. Membrane Transporter/Ion Channel
    Autophagy
  2. CFTR
    Autophagy
  3. Ataluren

Ataluren (Synonyms: PTC124)

Cat. No.: HY-14832 Purity: 99.12%
Handling Instructions

Ataluren (PTC124) is an orally available CFTR-G542X nonsense allele inhibitor.

For research use only. We do not sell to patients.

Ataluren Chemical Structure

Ataluren Chemical Structure

CAS No. : 775304-57-9

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10 mM * 1 mL in DMSO USD 66 In-stock
Estimated Time of Arrival: December 31
10 mg USD 60 In-stock
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50 mg USD 150 In-stock
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100 mg USD 240 In-stock
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200 mg USD 288 In-stock
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Customer Review

Based on 3 publication(s) in Google Scholar

Top Publications Citing Use of Products

    Ataluren purchased from MCE. Usage Cited in: Biopolymers. 2014 Apr;101(4):391-7.

    Western blot (α-FLAG) comparison of LexA+Gal4 Phe849pBpa expressed in the presence of increasing concentrations of PTC124. Additional details can be found in the Methods section.
    • Biological Activity

    • Protocol

    • Purity & Documentation

    • References

    • Customer Review

    Description

    Ataluren (PTC124) is an orally available CFTR-G542X nonsense allele inhibitor.

    IC50 & Target

    CFTR[1]

    In Vitro

    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].

    In Vivo

    Ataluren (PTC124) activity, optimized using nonsense-containing reporters, promotes dystrophin production in primary muscle cells from humans and mdx mice expressing dystrophin nonsense alleles, and rescues striated muscle function in mdx mice within 2-8 weeks of drug exposure. Ataluren (PTC124) is well tolerated in animals at plasma exposures substantially in excess of those required for nonsense suppression[2]. To induce nonsense suppression and increase PPT1 enzyme activity, the read-through drug Ataluren (PTC124) is given via intraperitoneal (i.p.) injection to male Cln1R151X mice at 2 months of age. These treatments are performed four times daily for 2 consecutive days in a proof-of-principle study. Used at 10 mg/kg, Ataluren (PTC124) increased PPT1 enzyme activity (P=0.0001 by unpaired t-test) and protein level (P=0.0014 by unpaired t-test) in the liver, but did not increase PPT1 enzyme activity or protein level in the cortex. This tissue-specific effect is likely due to the inability of Ataluren (PTC124) to breach the blood brain barrier (BBB), which decreased the bioavailability of Ataluren (PTC124) within the brain, and prevented Ataluren (PTC124) from reaching an efficacious concentration within the therapeutic window[3].

    Clinical Trial
    Molecular Weight

    284.24

    Formula

    C₁₅H₉FN₂O₃

    CAS No.

    775304-57-9

    SMILES

    O=C(C1=CC=CC(C2=NOC(C3=CC=CC=C3F)=N2)=C1)O

    Shipping

    Room temperature in continental US; may vary elsewhere.

    Storage
    Powder -20°C 3 years
      4°C 2 years
    In solvent -80°C 6 months
      -20°C 1 month
    Solvent & Solubility
    In Vitro: 

    DMSO : ≥ 52 mg/mL (182.94 mM)

    H2O : < 0.1 mg/mL (insoluble)

    *"≥" means soluble, but saturation unknown.

    Preparing
    Stock Solutions
    Concentration Solvent Mass 1 mg 5 mg 10 mg
    1 mM 3.5182 mL 17.5908 mL 35.1815 mL
    5 mM 0.7036 mL 3.5182 mL 7.0363 mL
    10 mM 0.3518 mL 1.7591 mL 3.5182 mL
    *Please refer to the solubility information to select the appropriate solvent.
    In Vivo:
    • 1.

      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

    • 2.

      Add each solvent one by one:  10% DMSO    90% corn oil

      Solubility: ≥ 2.5 mg/mL (8.80 mM); Clear solution

    *All of the co-solvents are provided by MCE.
    References
    Cell Assay
    [2]

    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].

    MCE has not independently confirmed the accuracy of these methods. They are for reference only.

    Animal Administration
    [3]

    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.

    MCE has not independently confirmed the accuracy of these methods. They are for reference only.

    References
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    Keywords:

    AtalurenPTC124PTC 124PTC-124CFTRAutophagyCystic fibrosis transmembrane conductance regulatorInhibitorinhibitorinhibit

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