CYSTIC FIBROSIS: A DRIVE FOR ACQUIRING KNOWLEDGE

Abstract

Cystic fibrosis (CF) detrimentally affects the pulmonary and digestive system of patients and only a few years ago had a life expectancy of a mere 40 years. However, due to strides made in CF research the life expectancy projection is now 58 years. Cystic fibrosis transmembrane conductance regulator (CFTR), an anion channel located on the apical membrane of epithelia, is directly responsible for the translocation of chloride and indirectly responsible for the paracellular translocation of water. A mutation in CFTR causes systemic hyperosmotic epithelial secretions. My thesis focuses on existing knowledge about CF with emphasis on the basic defect of the disease and the current standard of care. Recent and noteworthy papers were collected and synthesized. Current treatments were examined for specificity and success in reducing symptoms. CF research is making great advancements in individualized treatment of this disease which has vast variation from patient to patient due to the vast number of mutations that can affect CFTR. CF research is an excellent example of how targeting specific mechanisms of a disease can decrease morbidity in CF patients. Having comprehensive knowledge may help in the understanding necessary to continue focused basic research and alleviate the health problems associated with CF.