CFTR
CFTR (Cystic fibrosis transmembrane conductance regulator) is a membrane protein and chloride channel in vertebrates that is encoded by the CFTR gene. The CFTR gene codes for an ABC transporter-class ion channel protein that conducts chloride and thiocyanate ions across epithelial cell membranes. Mutations of the CFTR gene affecting chloride ion channel function lead to dysregulation of epithelial fluid transport in the lung, pancreas and other organs, resulting in cystic fibrosis.
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VX-809能增加突变型CFTR(F508del-CFTR)的成熟，并纠正常见于囊性纤维化的CFTR突变，EC50为0.1 μM。
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Ivacaftor是CFTR的增强剂，对G551D-CFTR和F508del-CFTR的EC50分别为100 nM和25 nM。
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PTC124 (Ataluren)选择性诱导核糖体通读，不影响正常的终止密码子，EC50为0.1 μM，可以治疗无义突变（如CFTR无义突变引起的囊性纤维病）造成的遗传性疾病。
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CP-466722是快速可逆的ATM激酶抑制剂。
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Rivaroxaban是一种口服具有直接作用的Factor Xa抑制剂，Ki值为0.4 nM，IC50值为0.7 nM。
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