PTC124 (Ataluren) selectively induces ribosomal read-through of premature but not normal termination codons, with EC50 of 0.1 M, may provide treatment for genetic disorders caused by nonsense mutations (e.g. CF caused by CFTR nonsense mutation).
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化学信息
名称 | PTC124c |
分子式 | C15H9FN2O3 |
分子量 | 284.24 |
Smile | c1ccc(c(c1)c2nc(no2)c3cccc(c3)C(=O)O)F |
InChiKey | OOUGLTULBSNHNF-UHFFFAOYSA-N |
InChi | InChI=1S/C15H9FN2O3/c16-12-7-2-1-6-11(12)14-17-13(18-21-14)9-4-3-5-10(8-9)15(19)20/h1-8H,(H,19,20) |
Cas号 | 775304-57-9 |
MDL | MFCD09864996 |
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