阿塔鲁伦
Ataluren (PTC124)
CAS: 775304-57-9
Molecular Formula: C15H9FN2O3
阿塔鲁伦 - Names and Identifiers
| Name | Ataluren (PTC124) |
| Synonyms | Ptc124 Ptc-124 PTC-124,ataluren Ataluren (PTC124) PTC124, 775304-57-9 Ataluren PTC 124 Ataluren 3-[5-(2-Fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid |
| CAS | 775304-57-9 |
阿塔鲁伦 - Physico-chemical Properties
| 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. |
阿塔鲁伦 - 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.
Last Update：2022-10-16 17:41:49
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View History
阿塔鲁伦
4-羟基-3-碘喹啉
ALPHA,ALPHA,ALPHA-三氟苯乙酮
L-Glutamic acid dibenzyl ester hydrochloride
N-[叔丁氧羰基]甘氨酰-L-脯氨酸
Hydroxy-2-phenylpropyl CarbaMate
替莫唑胺相关化合物B(USP)
Diethylene glycol methyl ether
NSC 27435
GDC-0927
4-羟基-3-碘喹啉
ALPHA,ALPHA,ALPHA-三氟苯乙酮
L-Glutamic acid dibenzyl ester hydrochloride
N-[叔丁氧羰基]甘氨酰-L-脯氨酸
Hydroxy-2-phenylpropyl CarbaMate
替莫唑胺相关化合物B(USP)
Diethylene glycol methyl ether
NSC 27435
GDC-0927