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- PTC-124 (Ataluren) | Avoid early termination caused by nonsense mutation
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- PTC-124 (Ataluren) | Avoid early termination caused by nonsense mutation
Product Description
PTC-124 (Ataluren) is an oxadiazole-based, orally available agent deisgned to selectively promote ribosomal readhtrough of premature stop codons, but not normal termination codons. [1] The minimal concentration of PTC124 showing observable readthrough was 0.01 - 0.1 uM (2.8 - 28 ng/ml), while maximal activity was observed at 3 uM (852 ng/ml). [2]
PTC-124 has been studied extensively in the treatment of cystic fibrosis and has shown good tolerability and efficacy in preclinical and clinical settings. [3, 4]
Technical information:
| Chemical Formula: | C15H9FN2O3 | |
| CAS #: | 775304-57-9 | |
| Molecular Weight: | 284.24 | |
| Purity: | >98% | |
| Appearance: | White | |
| Chemical Name: | 3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoic acid | |
| Solubility: | Up to 100 mM in DMSO | |
| Synonyms: | PTC-124, PTC124, PTC 124 |
Shipping Condition: The product is shipped in a glass vial at ambient temperature.
Storage condition: For longer shelf life, store solid powder at 4oC desiccated, or store DMSO solution at -20oC.
Reference:
| 1. | Sermet-Gaudelus et al., Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis. Am. J. Respir. Crit. Care Med. 2010, 182, 1262-1272. Pubmed ID: 20622033 |
| 2. | Welch et al., PTC124 targets genetic disorders caused by nonsense mutations. Nature, 2007, 447, 87-91. Pubmed ID: 17450125 |
| 3. | Du et al., PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model. Proc. Natl. Acad. Sci. 2008, 105(6), 2064-2069 Pubmed ID: 18272502 |
| 4. | Wilschanski et al., Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis. Eur. Respir. J. 2011, 38, 59-69. Pubmed ID: 17450125 |
Other Information:
Product Specification (pdf)
MSDS (pdf)
Certificate of Analysis is available upon request.
