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商品描述
ProductDescription
PTC-124(Ataluren)isanoxADIazole-based,orallyavailableagentdeisgnedtoselectivelypromoteribosomalreadhtroughofprematurestopcodons,butnotnormalterminationcodons.[1]TheminimalconcentrationofPTC124showingobservablereadthroughwas0.01-0.1uM(2.8-28ng/ml),whilemaximalactivitywasobservedat3uM(852ng/ml).[2]
PTC-124hasbeenstudiedextensivelyinthetreatmentofcysticfibrosisandhasshowngoodtolerABIlityandefficacyinpreclinicalandclinicalsettings.[3,4]
Technicalinformation:
| ChemicalFormula: | C15H9FN2O3 | |
| CAS#: | 775304-57-9 | |
| MolecularWeight: | 284.24 | |
| Purity: | >98% | |
| Appearance: | White | |
| ChemicalName: | 3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoicacid | |
| Solubility: | Upto100mMinDMSO | |
| Synonyms: | PTC-124,PTC124,PTC124 |
ShippingCondition:Theproductisshippedinaglassvialatambienttemperature.
Storagecondition:Forlongershelflife,storesolidpowderat4oCdesiccated,orstoreDMSOsolutionat-20oC.
Reference:
| 1. | Sermet-Gaudelusetal.,Ataluren(PTC124)inducescysticfibrosistransmembraneconductanceregulatorproteinexpressionandactivityinchildrenwithnonsensemutationcysticfibrosis.Am.J.Respir.Crit.CareMed.2010,182,1262-1272.PubmedID:20622033 |
| 2. | Welchetal.,PTC124targetsgeneticdisorderscausedbynonsensemutations.Nature,2007,447,87-91.PubmedID:17450125 |
| 3. | Duetal.,PTC124isanorallybioavailablecompoundthatpromotessuppressionofthehumanCFTR-G542XnonsensealleleinaCFmousemodel.Proc.Natl.Acad.Sci.2008,105(6),2064-2069PubmedID:18272502 |
| 4. | Wilschanskietal.,Chronicataluren(PTC124)treatmentofnonsensemutationcysticfibrosis.Eur.Respir.J.2011,38,59-69.PubmedID:17450125 |
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