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The development of a successful gene therapy has many stages, including preclinical testing in animal models and proof of principle clinical studies. A variety of diseases affect the lung, which are candidates for gene therapy; this review will mainly focus on the diseases that have attracted the most attention and have therefore yielded the most progress, namely lung cancer and the monogenic disorder cystic fibrosis. Knowledge gained from clinical studies could eventually be applied to more complex lung conditions such as acute respiratory distress syndrome and asthma. In addition, increased gene transfer efficiencies could be obtained by appropriate selection of the gene transfer vector and mode of delivery.

Original publication




Journal article


Cell Mol Life Sci

Publication Date





355 - 368


Animals, Cystic Fibrosis, Gene Transfer Techniques, Genetic Therapy, Genetic Vectors, Humans, Liposomes, Lung, Lung Diseases