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Cystic fibrosis (CF) a monogenic lethal disease and, therefore, ideally suited for the development of gene therapy. The first clinical trials were carried out shortly after cloning the CF gene in 1989. Since then, 25 trials have been carried out. Proof of principle for low-level airway gene transfer was established in most, but not all, trials. It is currently unclear whether current gene transfer efficiency will lead to improvements in clinically relevant endpoints such as inflammation or infection. In addition to addressing this important question, we and others are further improving airway gene transfer, by modifying existing and developing new gene transfer agents. Here, we describe pre-clinical methods related to assessing correction of the CF chloride transport defect.

Original publication

DOI

10.1007/978-1-59745-237-3_14

Type

Journal article

Journal

Methods Mol Biol

Publication Date

2008

Volume

433

Pages

229 - 242

Keywords

1-Methyl-3-isobutylxanthine, Animals, Cell Line, Cell Separation, Colforsin, Cystic Fibrosis Transmembrane Conductance Regulator, Epithelial Cells, Humans, Ion Transport, Luciferases, Mice, Respiratory System, Transfection