Orally bioavailable 284 D-1,2,4-oxadiazole. Promotes readthrough of premature translation termination codons. Targets genetic disorders caused by nonsense mutations. Shows no concomitant effects on normal termination or mRNA decay.
Product Details
| Alternative Name: | 3-[5-(2-Fluorophenyl)-1,2,4-oxadiazol-3-yl]-benzoic acid |
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| Formula: | C15H9FN2O3 |
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| MW: | 284.2 |
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| CAS: | 775304-57-9 |
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| Purity: | ≥95% |
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| Identity: | Identity determined by MS, 1H- and 13C-NMR. |
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| Appearance: | White to off-white solid. |
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| Solubility: | Soluble in DMSO (5mg/ml). |
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| Shipping: | Ambient |
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| Long Term Storage: | -20°C |
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| Handling: | Protect from light. |
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| Regulatory Status: | RUO - Research Use Only |
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Product Literature References
Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial: E. Kerem, et al.; Lancet
372, 719 (2008),
Abstract;
PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model: M. Du, et al.; PNAS
105, 2064 (2008),
Abstract;
PTC124 targets genetic disorders caused by nonsense mutations: E.M. Welch, et al.; Nature
447, 87 (2007),
Abstract;
