1.For some materials, we could supply free Samples for testing at first..
2.MOQ: 1g or 5g
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What is CAS 775304-57-9 Ataluren Powder?
| Product Name: |
Ataluren |
| Synonyms: |
Ptc124;Ptc-124;PTC-124,ataluren;Ataluren (PTC124);Ataluren PTC 124 Ataluren;Ataluren, >=98%;PTC124, 775304-57-9;3-[5-(2-Fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid |
| CAS: |
775304-57-9 |
| MF: |
C15H9FN2O3 |
| MW: |
284.24 |
| EINECS: |
|
| Product Categories: |
Inhibitors;Ribosome binding agent;API |
| Mol File: |
775304-57-9.mol |
1.Ataluren is a drug marketed under the trade name Translarna® which was developed by PTC Therapeutics and approved by the European Union in May 2014 for the treatment of Duchenne's muscular dystrophy (DMD) and potentially other genetic disorders. Ataluren renders ribosomes less sensitive to premature stop or 'read-through' codons, which are thought to be beneficial in diseases such as DMD and cystic fibrosis.
2. Nonsense mutations create a premature termination of mRNA translation and have been implicated in various genetic disorders, including muscular dystrophy and cystic fibrosis. PTC-124 is a nonaminoglycoside that has been reported to selectively induce ribosomes to read through premature nonsense stop signals on mRNA, thus allowing the production of full length, functional proteins. In a mouse model of cystic fibrosis caused by nonsense mutations, PTC-124 treatment (60 mg/kg s.c. injection or 0.3-0.9 mg/ml orally) has been shown to restore cystic fibrosis transmembrane conductance regulator (CFTR) protein expression and function. The target activity of PTC-124 was initially evaluated by firefly luciferase reporter cell-based nonsense codon assay (IC50 = 7 nM); however, subsequent assessments using a Renilla reniformis luciferase reporter have failed to produce nonsense codon suppression activity. Thus, while PTC-124 is in clinical testing in patients with nonsense mutations within the CFTR or dystrophin genes, controversy surrounds its exact mechanism of action.
3. Nonsense mutations create a premature termination of mRNA translation and have been implicated in various genetic disorders, including muscular dystrophy and cystic fibrosis. PTC-124 is a nonaminoglycoside that has been reported to selectively induce ribosomes to read through premature nonsense stop signals on mRNA, thus allowing the production of full-length, functional proteins. In a mouse model of cystic fibrosis caused by nonsense mutations, PTC-124 treatment (60 mg/kg s.c. injection or 0.3-0.9 mg/ml orally) has been shown to restore cystic fibrosis transmembrane conductance regulator (CFTR) protein expression and function. The target activity of PTC-124 was initially evaluated by firefly luciferase reporter cell-based nonsense codon assay (IC50 = 7 nM); however, subsequent assessments using a Renilla reniformis luciferase reporter have failed to produce nonsense codon suppression activity. Thus, while PTC-124 is in clinical testing in patients with nonsense mutations within the CFTR or dystrophin genes, controversy surrounds its exact mechanism of action.[Cayman Chemical]
4. PTC-124 is a nonaminoglycoside that has been reported to induce ribosomes to read through premature nonsense stop signals on mRNA, allowing the production of full-length functional proteins.
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