| 产品详情 |
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| Product Name | PTC-124 |
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| Description | Purity >98%. 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. |
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| Size | n/a |
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| Concentration | n/a |
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| Applications | n/a |
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| Other Names | 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]-benzoic acid, Ataluren |
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| Gene, Accession, CAS # | CAS: 775304-57-9 |
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| Catalog # | LS-H9538 |
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| Order / More Info | PTC-124 from LIFESPAN BIOSCIENCES INC. |
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| Product Specific References | n/a |
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