ABSTRACT
I. Issues for the Success of CF Gene Therapy While there are an array of functions and conflicting theories have been put forth to explain the pathophysiology of cystic fibrosis (CF) as a result of the absence of the cystic fibrosis transmembrane regulator (CFTR), it is widely accepted that a predilection to infection and an exaggerated and sustained inflammatory response to these bacterial, viral, and fungal agents in the lung leading to progressive tissue damage is a main feature of the CF phenotype. Since the majority of CF related mortality has been associated with the lung, this has made the disease a prime candidate for pulmonary gene therapy by gene augmentation.
