Abstract EN:

Cystic Fibrosis, the most common lethal genetic disease in Caucasian populations, is characterized by its great genotypic and phenotypic variability. This disease mainly affects both digestive and pulmonary functions, and the progressive respiratory degradation ultimately leads to death. Although many therapeutic strategies have emerged for the past decade, efficient treatments to cure permanently this disease are not available yet. Preventing the genetic defect would be the ideal way to achieve this goal. Since the CFTR gene, responsible for the disease was identified in 1989, gene therapy has become the major hope for CF patients. Many gene vectors, such as adenoviral-derived vectors and synthetic reagents have been evaluated to deliver therapeutic genes to the airways and numerous clinical trials have been initiated to assess both their safety and efficacy. Professor Férec, in Brest, has decided to penetrate this field by testing and developing a new family of cationic lipids for gene transfer to the lungs. Biodistribution of these reagents as well as kinetics of expression of the reporter genes were studied in vivo in mice following different ways of administration by various techniques, including imaging-based systems. Although we and others have collected a broad range of data including parameters influencing transfection efficiency and tissue targeting, a long and hard work is still to be done before gene therapy strategies can be routinely used as a common treatment to cure the CF disease.