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- W151503897 abstract "Cystic fibrosis (CF) is characterised by respiratory and pancreatic deficiencies that stem from the loss of fully functional CFTR (CF transmembrane conductance regulator) at the membrane of epithelial cells. Current treatment modalities aim to delay the deterioration in lung function, Which is mostly responsible for the relatively short life expectancy of CF sufferers; however none have so far successfully dealt with the underlying molecular defect. Novel pharmacological approaches to ameliorate the lack of active CFTR in respiratory epithelial cells are beginning to address more of the pathophysiological defects caused by CFTR mutations. However, CFTR gene replacement by gene therapy remains the most likely option for addressing the basic defects, including ion transport and inflammatory functions of CFTR. In this chapter, We will review the latest preclinical and clinical advances in pharmacotherapy and gene therapy for CF lung disease." @default.
- W151503897 created "2016-06-24" @default.
- W151503897 creator A5032599226 @default.
- W151503897 creator A5036517483 @default.
- W151503897 creator A5069237359 @default.
- W151503897 date "2010-01-01" @default.
- W151503897 modified "2023-09-25" @default.
- W151503897 title "Gene therapy for cystic fibrosis lung disease" @default.
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