Emerging drug treatments for cystic fibrosis.

摘要: Cystic fibrosis (CF) is one of the most common life-shortening inherited disorders. Mutations in cystic transmembrane regulator (CFTR) gene disrupt localisation and function cAMP-mediated chloride channel. Most morbidity mortality arise from lung disease which characterised by excessive inflammation chronic infection. Research into mechanisms wild-type mutant CFTR biogenesis suggest that multiple drug targets can be identified. This review explores current understanding nature different forms potential for repair channel defect. High-throughput screening, pharmacogenomics proteomics bring recent technological advances to field.