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PTC124

 
ALX-430-158-M002 2 mg 210.00 USD
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Orally bioavailable 284 D-1,2,4-oxadiazole. Promotes readthrough of premature translation termination codons. Targets genetic disorders caused by nonsense mutations. Shows no concomitant effects on normal termination or mRNA decay.

Product Specification

Alternative Name:3-[5-(2-Fluorophenyl)-1,2,4-oxadiazol-3-yl]-benzoic acid
 
Formula:C15H9FN2O3
 
MW:284.2
 
CAS:775304-57-9
 
Purity:≥95%
 
Identity:Identity determined by MS, 1H- and 13C-NMR.
 
Appearance:White to off-white solid.
 
Solubility:Soluble in DMSO (5mg/ml).
 
Shipping:Ambient
 
Long Term Storage:-20°C
 
Handling:Protect from light.
 
430-158
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430-158

Product Literature References

Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial: E. Kerem, et al.; Lancet 372, 719 (2008), Abstract;
PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model: M. Du, et al.; PNAS 105, 2064 (2008), Abstract;
PTC124 targets genetic disorders caused by nonsense mutations: E.M. Welch, et al.; Nature 447, 87 (2007), Abstract;

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