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Gene therapy has been proposed for a wide range of human diseases but few have received the level of attention over such a prolonged period as cystic fibrosis (CF) with over 20 clinical studies undertaken. Following a 10-year interval, clinical trials of an aerosolisable non-viral gene transfer agent have recently been initiated by researchers in the United Kingdom. Here we review the rationale and requirements for effective gene therapy for CF lung disease. The previous non-viral gene therapy trials are discussed and the prospects for the current leading non-viral formulations for CF gene therapy are considered. Factors affecting the selection and design of the plasmid DNA molecule, likely to be of central importance to clinical efficacy, are reviewed and we describe the potential merits of the formulation that has been selected for the forthcoming UK trials.

Original publication

DOI

10.1517/14712590903055029

Type

Journal article

Journal

Expert Opin Biol Ther

Publication Date

08/2009

Volume

9

Pages

991 - 1003

Keywords

Aerosols, Animals, Clinical Trials as Topic, CpG Islands, Cystic Fibrosis, DNA, Gene Transfer Techniques, Genetic Therapy, Genetic Vectors, Humans, Lipids, Plasmids, Polymers, Promoter Regions, Genetic