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Ataluren (PTC124)(CAS#775304-57-9) Featured Image
  • Ataluren (PTC124)(CAS#775304-57-9) 1

Ataluren (PTC124)(CAS#775304-57-9)

Chemical Property:

Molecular Formula C15H9FN2O3
Molar Mass 284.24
Density 1.379
Melting Point 241 – 242°C
Boling Point 503.7±60.0 °C(Predicted)
Solubility Soluble in DMSO (57 mg/ml), water (<1 mg/mL), and ethanol (<1 mg/mL).
Appearance White to off-white solid.
Color White to Off-White
pKa 3.58±0.10(Predicted)
Storage Condition Refrigerator
Use A firefly luciferase inhibitor and an inducer of ribosomal read-through of mRNAs.
In vitro study Compared with Gentamicin, which is only active at higher concentrations, PTC124 is a more potent inhibitor of nonsense, and the read-through stimulation is 4-15 times stronger than that of the control group. PTC124(0.01-3 μm) acted on HEK293 cells containing LUC-190 nonsense alleles to promote read-through of three nonsense codons in a dose-dependent manner, with the highest read-through in UGA, this is followed by UAG and then UAA, but does not suppress multiple proximal nonsense codons. Similar to Gentamicin, PTC124 is most active at the pyrimidine after the nonsense codon (especially cytosine, C). Consistent with stable cell line reporting experiments, PTC124 (17 μm) significantly promoted dystrophin production in patients with Duchenne muscular dystrophy (DMD) or in mdx mice expressing the dystrophin nonsense allele. PTC124 selectively promotes read-through of the ribosomal premature stop codon rather than the normal stop codon, even at concentrations significantly higher than required to achieve maximum activity.
In vivo study Because of functional restoration of dystrophin production, PTC124 was administered orally, intraperitoneally, or a combination of both for 2-8 weeks to partially alleviate functional strength deficits in dystrophic muscles of mdx mice, results The effect on extensor digitorum longus (EDL) played a local protective effect on contractile injury and significantly reduced serum creatine kinase values. PTC124 subcutaneously or orally treated Cftr-/-mice expressing the human CFTR-G542X transgene at a dose of ~ 60 mg/kg inhibited the G542X nonsense mutation in a dose-dependent manner, resulting in human (h) significant restoration of CFTR protein expression and function without any effect on nonsense-mediated mRNA degradation (NMD) or other aspects of mRNA stability. Treatment of Cftr +/+ mice with PTC124 at a dose of 60 mg/kg observed a 29% recovery of short-circuit current stimulated by normal intestinal transepithelial cAMP, a significant advantage over Gentamicin.

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Introduction

Ataluren (PTC124) selectively induces ribosome read-through without affecting the normal stop codon. The EC50 is 0.1 μM, which can treat nonsense mutations (nonsense mutations) (such as cystic fibrosis (CF) caused by CFTR nonsense mutations) Genetic diseases caused by disease.

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