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My Cart> Small Molecule Compounds > Inhibitors & Antagonists & Agonists > Membrane Transporter & Ion Channel > PTC124CAS：775304-57-9
Molecular Formula：C15H9FN2O3
Molecular Weight：284.24
Storage：Powder:2-8℃，2 years;Insolvent(Mother Liquid):-20℃，6 months;-80℃，1 year
Purity：≥98%
Appearance：White to off-white Solid
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| CAS | 775304-57-9 |
| Name | PTC124 |
| Molecular Formula | C15H9FN2O3 |
| Molecular Weight | 284.24 |
| Solubility | Soluble in DMSO |
| Purity | ≥98% |
| Appearance | White to off-white Solid |
| Storage | Powder:2-8℃，2 years;Insolvent(Mother Liquid):-20℃，6 months;-80℃，1 year |
| MDL | MFCD09864996 |
| SMILES | O=C(C1=CC=CC(C2=NOC(C3=CC=CC=C3F)=N2)=C1)O |
| Target Point | CFTR |
| Passage | Membrane Transporter&Ion Channel |
| Background | PTC124 is a CFTR-G542X nonsense allele inhibitor. |
| Biological Activity | Ataluren是 CFTR-G542X 无义等位基因抑制剂。[1-3] |
| Data Literature Source | [1]. Pettit RS， et al. CFTR Modulators for the Treatment of Cystic Fibrosis. P T. 2014 Jul; 39 (7) :500-11. [2]. Welch EM， et al. PTC124 targets genetic disorders caused by nonsense mutations. Nature， 2007， 447 (7140) ， 87-91. [3]. Miller JN， et al. The novel Cln1 (R151X) mouse model of infantile neuronal ceroid lipofuscinosis (INCL) for testing nonsense suppression therapy. Hum Mol Genet. 2015 Jan 1; 24 (1) :185-96. |
| Unit | Piece |
| Specification | 10mg 50mg |
Remark：These protocols are for reference only. Solarbio does not independently validate these methods.
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