PTC124
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| PTC124 | |
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| IUPAC name | 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid |
| Identifiers | |
| CAS number | |
| PubChem | |
| SMILES | C1=CC=C(C(=C1)C2=NC(=NO2)C3=CC(=CC=C3)C(=O)O)F |
| Properties | |
| Molecular formula | C15H9FN2O3 |
| Molar mass | 284.241963 |
| Except where noted otherwise, data are given for materials in their standard state (at 25 °C, 100 kPa) Infobox disclaimer and references |
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PTC124 is a novel small-molecular agent that makes ribosomes less sensitive to stop codons. However, it is not equally effective with every stop codon, working best on the sequence 'UGA' [1].
It has been investigated for use against conditions caused by nonsense mutations[1][2], such as Duchenne muscular dystrophy and cystic fibrosis.
PTC124 is scheduled to enter the final phase of clinical trials in 2007.[3]
It has been developed by PTC Therapeutics.[4]
[edit] References
- ^ a b Welch EM, Barton ER, Zhuo J, et al (2007). "PTC124 targets genetic disorders caused by nonsense mutations". Nature 447 (7140): 87–91. doi:. PMID 17450125.
- ^ Hirawat S, Welch EM, Elfring GL, et al (2007). "Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers". Journal of clinical pharmacology 47 (4): 430–44. doi:. PMID 17389552.
- ^ Daily pill to beat genetic diseases - Times Online. Retrieved on 2007-10-23.
- ^ Hamed SA (2006). "Drug evaluation: PTC-124--a potential treatment of cystic fibrosis and Duchenne muscular dystrophy". IDrugs : the investigational drugs journal 9 (11): 783–9. PMID 17096300.
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