Nanotechnology approach for treatment of cystic fibrosis.

摘要

Cystic fibrosis is a lethal, hereditary disease, caused by mutations in the gene cystic fibrosis transmembrane conductance regulator (CFTR) where it affects a cAMP-activated chloride (Cl-) channel and results in impaired ion and water transport, accumulation of viscous mucus in the airway, thereby leads to bacterial colonization. Frequent infections and inflammation result in an obstructive pulmonary disease. Similar conditions in the pancreas lead to pancreatic insufficiency. Several approaches have been designed to improve transepithelial ion transport in CF patients. These approaches involve correction of the CFTR mutation, potentiation of CFTR channel, and/or stimulation of alternative chloride channels. The purpose of this thesis involve using a nanotechnology to correct the defective ion transport in epithelial cells from CF patient. The first aim of the project is comparative study to evaluate the effect of using corrector and/or potentiator agent to restore the ion channel activity. The second aspect is nanotechnology-based delivery system that enhance drug delivery to the lung cells to treat pulmonary cystic fibrosis.