[Gene therapy in cystic fibrosis: molecular and cellular aspects]

Gene therapy has become a potential treatment for Cystic Fibrosis (CF) when the CFTR gene responsible for the disease was isolated in 1989. As premature death is still the norm, the demand is now for a dramatically improved therapy. The pulmonary manifestations of CF being life-limiting, many gene therapy studies have focused on gene transfer via various vectors into airway epithelial cells, namely in murine models of CF. These preclinical studies led to the first clinical trials, which showed that many hurdles still do exist and that more efficient vector systems need to be developed. Both a remarkable scientific effort and a close collaboration between scientists and clinicians are highly necessary in order to move from the present stage of "cautious optimism" toward the ultimate goal: a cure for CF.