Shelf life (days): 1460.0
Formulation: A crystalline solid
Formal Name: 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]-benzoic acid
Purity: ≥98%
Formula Markup: C15H9FN2O3
Formula Weight: 284.3
CAS Number: 775304-57-9
Notes: Nonsense mutations create a premature termination of mRNA translation and have been implicated in various genetic disorders, including muscular dystrophy and cystic fibrosis.{27189} 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.{27189} 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.{27187} 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.{27188} 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.{27189,27188,27191,27190}
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