Zegarra-Moran Olga, Galietta Luis J V
U.O.C. Genetica Medica, Istituto Giannina Gaslini, Via Gerolamo Gaslini 5, 16147, Genoa, Italy.
Cell Mol Life Sci. 2017 Jan;74(1):117-128. doi: 10.1007/s00018-016-2392-x. Epub 2016 Oct 4.
CFTR protein is an ion channel regulated by cAMP-dependent phosphorylation and expressed in many types of epithelial cells. CFTR-mediated chloride and bicarbonate secretion play an important role in the respiratory and gastrointestinal systems. Pharmacological modulators of CFTR represent promising drugs for a variety of diseases. In particular, correctors and potentiators may restore the activity of CFTR in cystic fibrosis patients. Potentiators are also potentially useful to improve mucociliary clearance in patients with chronic obstructive pulmonary disease. On the other hand, CFTR inhibitors may be useful to block fluid and electrolyte loss in secretory diarrhea and slow down the progression of polycystic kidney disease.
囊性纤维化跨膜传导调节因子(CFTR)蛋白是一种受环磷酸腺苷(cAMP)依赖性磷酸化调节的离子通道,在多种上皮细胞中表达。CFTR介导的氯离子和碳酸氢根分泌在呼吸和胃肠系统中发挥重要作用。CFTR的药理调节剂是治疗多种疾病的有前景的药物。特别是,校正剂和增强剂可能恢复囊性纤维化患者CFTR的活性。增强剂对于改善慢性阻塞性肺疾病患者的黏液纤毛清除功能也可能有用。另一方面,CFTR抑制剂可能有助于阻止分泌性腹泻中的液体和电解质流失,并减缓多囊肾病的进展。