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  • Posenacaftor sodium
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Posenacaftor sodium

Posenacaftor (PTI-801) sodium 是一种囊性纤维化跨膜调节蛋白 (CFTR) 调节剂,可纠正 CFTR 蛋白的折叠和运输。Posenacaftor sodium 用于囊性纤维化 (CF) 的研究。

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  • 货号: ajcx33888
  • CAS: 2095064-06-3
  • 别名: PTI-801 sodium
  • 分子式: C27H26NNaO5
  • 分子量: 467.49
  • 纯度: >98%
  • 溶解度: DMSO : 200 mg/mL (427.82 mM; Need ultrasonic)
  • 储存: Store at -20°C
  • 库存: 现货

Background

Posenacaftor (PTI-801) sodium is a cystic fibrosis transmembrane regulator (CFTR) protein modulator that corrects the folding and trafficking of CFTR protein. Posenacaftor sodium is used for the research of cystic fibrosis (CF)[1].


Cystic fibrosis (CF) is an autosomal recessive disorder, caused by mutations of the cystic fibrosis transmembrane conductance regulator (CFTR)[1].CFTR is a cAMP-regulated chloride channel that is primarily located at the apical membrane of epithelial cells.Mutations in the CFTR gene lead to the production of a defective and misfolded CFTR protein, and impairs the flow of ions in and out of cells[1].Posenacaftor is a CFTR corrector, correctors are always designed to fix and restore the function of the defective CFTR protein. The corrected CFTR then moves to the cell surface, where it functions as a chloride channel and helps maintain the right balance of fluid in the airways[2].


[1]. Benjamin Kopp, et al. Compositions et procédés pour améliorer la fonction cftr dans des cellules affectées par la fibrose kystique. Patent WO2019156946.
[2]. Posenacaftor (PTI-801)

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