4-[[9-氯-7-(2-氟-6-甲氧基苯基)-5H-嘧啶并[5,4-D][2]苯并氮杂卓-2-基]氨基]-2-甲氧基苯甲酸

溶解度:Soluble in DMSO (5mg/ml)

储存条件:-20C 2 years

生物活性

Treat Cystic Fibrosis and Duchenne and Becker Muscular Dystrophy (nmDMD/BMD). It shows promising result in Phase 2. 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.

参考文献

1��PTC124 targets genetic disorders caused by nonsense mutations: E.M. Welch, et al.; Nature 447, 87 (2007) 2��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) 3��Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial: E. Kerem, et al.; Lancet 372, 719 (2008)