PTC-124

A cell-permeable, non-toxic, orally bioavailable oxadiazole compound that is reported to promote read-through of premature, but not normal, translation termination codons and thereby suppress premature termination in protein synthesis caused by nonsense mutations (in ~10 to 100 nM with maximal activity reported at 3 µM). Promotes the suppression of nonsense alleles of the dystrophin gene in primary human muscle cells and in a mouse model of Duchenne muscular dystrophy (mdx). Also reported to rescue striated muscle function in mdx mice with a noticeable reduction in their serum creatine kinase values. Subcutaneous or oral administration of PTC124 to a mice expressing human CFTR-G542X transgene is shown to suppress G542X nonsense mutation and restore hCFTR function and cAMP-stimulated chloride channel activity (60 mg/kg for 3 weeks). Also acts as a potent and reversible inhibitor of purified firefly luciferase (FLuc; IC₅₀ ~ 10 nM) and protects it from tryptic degradation, but does not affect Renilla reniformis luciferase (RLuc).

Cell permeable: yes

Primary Target
PTC

Secondary Target
firefly luciferase

Toxicity: Standard Handling (A)

Following reconstitution, aliquot and freeze (-20°C). Stock solutions are stable for up to 6 months at -20°C.

McElroy, S.P., et al. 2013. PLoS Biol.11, e1001593.
Auld, D.S., et al. 2010. Proc. Natl. Acad. Sci. USA.107, 4878.
Auld, D.S., et al. 2009. Proc. Natl. Acad. Sci. USA.106, 3585.
Du, M., et al. 2008. Proc. Natl. Acad. Sci. USA.105, 2064.

Welch, E.M., et al. 2007. Nature.447, 87.

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