Ataluren

SKU: orb1307406

Description

Ataluren

Research Area

Cell Biology, Pharmacology & Drug Discovery

Key Properties

• CAS Number: 775304-57-9
• MW: 284.24
• Purity: 99.45%
• Formula: C₁₅H₉FN₂O₃
• SMILES: FC1=C(C2=NC(=NO2)C3=CC(C(O)=O)=CC=C3)C=CC=C1
• Target: Autophagy,CFTR
• Solubility: H2O:< 1 mg/mL (insoluble or slightly soluble);10% DMSO+90% Corn Oil:2.5 mg/mL (8.8 mM);Ethanol:< 1 mg/mL (insoluble or slightly soluble);DMSO:69 mg/mL (242.75 mM)

Bioactivity

• In Vivo:
  0.01-3 μM PTC124 promoted dose-dependent through-reading of All three nonsense codons in HEK293 cells containin the LUC-190 nonsense allele, wit the highest readings at UGA, followed by UAG, and then UAA, but it did not inhibit multiple proximal nonsense codons. 17 μM PTC124 was consistent wit the Experimental assay reported fo the stable cell line, an the results of PTC124 (17 PTC124 (17 μM) promotes significant production of myotrophic proteins in Primary myoblasts from patients with Duchenne-type muscular dystrophy (DMD) or MDXMDX mice expressin the nonsense allele of myotrophic dystrophic protease.PTC124 selectively promote the readthrough of ribosomal premature termination codons, but not normal termination codons, even at concentration substantially higher than those required to achieve maximal activity. PTC124 is a more potent nonsense inhibitor than gentamicin, which is active only at higher concentration , and shows a 4- to 15-fold stimulation of read-through relative to the control.PTC124, similar to gentamicin, is most active at pyrimidines (especially cytosine, C) following nonsense codons.
• In Vitro:
  0.01-3 μM PTC124 promoted dose-dependent through-reading of All three nonsense codons in HEK293 cells containin the LUC-190 nonsense allele, wit the highest readings at UGA, followed by UAG, and then UAA, but it did not inhibit multiple proximal nonsense codons. 17 μM PTC124 was consistent wit the Experimental assay reported fo the stable cell line, an the results of PTC124 (17 PTC124 (17 μM) promotes significant production of myotrophic proteins in Primary myoblasts from patients with Duchenne-type muscular dystrophy (DMD) or MDXMDX mice expressin the nonsense allele of myotrophic dystrophic protease.PTC124 selectively promote the readthrough of ribosomal premature termination codons, but not normal termination codons, even at concentration substantially higher than those required to achieve maximal activity. PTC124 is a more potent nonsense inhibitor than gentamicin, which is active only at higher concentration , and shows a 4- to 15-fold stimulation of read-through relative to the control.PTC124, similar to gentamicin, is most active at pyrimidines (especially cytosine, C) following nonsense codons.
• Cell Research:
  PTC124 (Ataluren) is prepared in DMSO and stored, and then diluted with appropriate medium (DMSO 1%) before us. 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 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 s andard 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 softwar.

Storage & Handling

• Storage: Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice/Shipping at ambient temperature.
• Disclaimer: For research use only

Alternative Names

inhibit, Inhibitor, Autophagy, Ataluren, CFTR, Cystic fibrosis transmembrane conductance regulator, PTC 124, PTC124, PTC-124

Compound Identifiers

PubChem CID

11219835