1. Membrane Transporter/Ion Channel
  2. CFTR

PTC124 (Synonyms: Ataluren)

Cat. No.: HY-14832 Purity: 99.88%
Data Sheet SDS Handling Instructions

PTC124 targets nonsense mutations, which insert a termination codon in the middle of the CFTR gene.

For research use only. We do not sell to patients.
PTC124 Chemical Structure

PTC124 Chemical Structure

CAS No. : 775304-57-9

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10 mM * 1 mL in DMSO $55 In-stock
10 mg $50 In-stock
50 mg $125 In-stock
100 mg $200 In-stock
200 mg $240 In-stock
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Customer Review

    PTC124 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.

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    • Biological Activity

    • Protocol

    • Technical Information

    • Purity & Documentation

    • References

    Description

    PTC124 targets nonsense mutations, which insert a termination codon in the middle of the CFTR gene.

    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]. PTC124 (Ataluren)-a new chemical entity that selectively induces ribosomal readthrough of premature but not normal termination codons[2].

    In Vivo

    PTC124 (Ataluren) 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. 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 PTC124 (Ataluren) 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, 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 PTC124 to breach the blood brain barrier (BBB), which decreased the bioavailability of PTC124 within the brain, and prevented PTC124 from reaching an efficacious concentration within the therapeutic window[3].

    References
    Preparing Stock Solutions
    Concentration Volume 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.
    Cell Assay
    [2]

    PTC124 (Ataluren) is prepared in DMSO and stored, and then diluted with appropriate medium (DMSO 1%) before use[2].

    Duplicate samples of HEK293 cells harbouring LUC-190 (UGA) are incubated in the presence of 5 μM 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]

    PTC124 (Ataluren) is dissolved in PBS containing DMSO and 2-hydroxypropyl-β-cyclodextrin (22%) (Mice)[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 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
    Molecular Weight

    284.24

    Formula

    C₁₅H₉FN₂O₃

    CAS No.

    775304-57-9

    Storage
    Powder -20°C 3 years
      4°C 2 years
    In solvent -80°C 6 months
      -20°C 1 month
    Shipping

    Room temperature in continental US; may vary elsewhere

    Solvent & Solubility

    DMSO: ≥ 52 mg/mL

    * "<1 mg/mL" means slightly soluble or insoluble. "≥" means soluble, but saturation unknown.

    Purity: 99.88%

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    Inquiry Information

    Product Name:
    PTC124
    Cat. No.:
    HY-14832
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