10.1 Gene Therapy for Inherited Diseases of Lung

Gene therapy was originally developed as an approach for treatment of classic genetic diseases. The transfer of a normal copy of a single defective gene would compensate for the damaged gene and revert the disease. Recently it was used to treat other disease such as the lung diseases. There are three genetically controlled diseases of the lung due to a mutation at a single gene. These diseases are cystic fibrosis (CF), a1-antitrypsin deficiency (µ 1-ATD) and surfactant protein B (SF-B) deficiency.