Ataluren

Data Sheet For research use only. Not for human use.

Cat. No. ：BCP01756CAS No. ：775304-57-9Purity：98%

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CAS No.	775304-57-9	Cat. No.	BCP01756
Name	Ataluren
Synonyms	PTC-124;PTC 124;PTC124;
SMILES
Chemical Name
Formula	C15H9FN2O3	M. Wt	284.24
Purity	98%	Storage	Store at 4-8°C
Description	Compared with Gentamicin which is only active at much higher concentrations, PTC124 is a more potent nonsense-suppressing agent and exhibits 4- to 15-fold stimulation of read-through relative to controls. PTC124 (0.01-3 μM) promotes dose-dependent read-through of all three nonsense codons in HEK293 cells harboring LUC-190 nonsense alleles with the highest read-through at UGA, followed by UAG and then UAA, but it does not suppress multiple proximal nonsense codons. Like Gentamicin, PTC124 is most active when a pyrimidine (in particular cytosine, C) follows the nonsense codon. Consistent with the stable cell line reporter assay, PTC124 (17 μM) promotes significant production of dystrophin in primary muscle cells from Duchenne muscular dystrophy (DMD) patients or MDXMDX mice expressing dystrophin nonsense alleles. PTC124 selectively promotes ribosomal read-through of premature termination but not normal termination codons, even at concentrations substantially greater than the values achie