PTC124 Inhibitor CAS No. 775304-57-9

Cat. No.	Name	Size	Price
KI0006	PTC124	10 mg	$150
	PTC124	25 mg	$300
	PTC124	100 mg	$330
	PTC124	100 mg	$592

Chemical Characteristic

Product Name	PTC124
Synonyms	Ataluren
CAS No.	775304-57-9
Molecular Weight	284.24
Formula	C₁₅H₉FN₂O₃
Chemical Name	3-[5-(2-Fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid
Smiles	c1c(cccc1C(=O)O)c1noc(n1)c1ccccc1F
Chemical Structure
Documents	HNMR- DMSO LCMS

Biological activities

PTC124 is a potent firefly luciferase (FLuc) inhibitor. The IC50 of with PTC124 is 7 nM against FLuc. PTC124 is used as a potent nonsense-suppressing agent. PTC124 is a new chemical entity that selectively induces ribosomal read-through of premature but not normal termination codons. PTC124 (2 µM) slows the proteolysis half-life of firefly luciferase by about 2-fold but shows no effect on the proteolysis half-life of Renilla reniformis luciferase (RLuc).^[1] PTC124 promotes dose-dependent read-through of all three nonsense codons (UAA, UAG or UGA) in stable HEK293 cells harbouring LUC-190 nonsense alleles. The minimal concentration of PTC124 showing discernable readthrough is 0.01-0.1 µM (2.8-28 ng/mL), whereas the concentration promoting maximal activity is approximately 3 µM (852 ng/mL). PTC124 promotes suppression of human and mouse nonsense alleles of the dystrophin gene. In primary cell cultures derived from muscle biopsies, 17 µM (5 µg/mL) of PTC124 has the most efficient read-through and there is no further increase at 10 µg/mL. The ratio of dystrophin:myosin obtained at 5 µg/mL of PTC124 is 40-60% of normal. For the samples from a model of Duchenne muscular dystrophy (DMD), the dystrophin:myosin ratio is approximately 35% of normal at 10 µg/mL. In the model of Duchenne muscular dystrophy, either intraperitoneal injections or oral dosing alone of PTC124 results in partial protection against contraction-induced injury in the extensor digitorum longus (EDL) muscles.^[2] In a mouse model for cystic fibrosis (CF), s.c. injection or oral administration of PTC124 (15, 30, or 60 mg/kg) to Cftr -/- mice expressing a human cystic fibrosis transmembrane conductance regulator (CFTR)-G542X transgene suppress the G542X nonsense mutation and restore a significant amount of human (h)CFTR protein and function. PTC124 treatment also results in the appearance of hCFTR protein at the apical surface of intestinal glands in Cftr -/- hCFTR-G542X mice. In addition, functional assays demonstrate that PTC124 treatment restores 24-29% of the average cAMP-stimulated transepithelial chloride currents observed in wild-type mice.^[3]

Protocols

Stock solutions of PTC124 (6 mM) are prepared in 100% DMSO.^[2]

References

[1] Auld DS, et al. Mechanism of PTC124 activity in cell-based luciferase assays of nonsense codon suppression. Proc Natl Acad Sci U S A. 2009, 106(9): 3585-3590. [2] Welch EM, et al. PTC124 targets genetic disorders caused by nonsense mutations. Nature. 2007, 447(7140): 87-91. [3] Du M, et al. PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model. Proc Natl Acad Sci USA. 2008, 105(6): 2064-2069.

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