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Gene therapy was suggested as a potential treatment for cystic fibrosis (CF), even before the identification of the CFTR gene. Initial enthusiasm has been tempered as it became apparent that reintroduction of the CFTR gene into the cells of the lung is more difficult than anticipated. Here, we review the major gene delivery vectors evaluated clinically, and suggest that advances in either plasmid DNA design and/or hybrid lentivirus biology may finally facilitate lung gene transfer with efficiencies sufficient for CF gene therapy to offer clinical benefit.

Original publication

DOI

10.1136/thoraxjnl-2014-205835

Type

Journal article

Journal

Thorax

Publication Date

10/2014

Volume

69

Pages

962 - 964

Keywords

Cystic Fibrosis, Cystic Fibrosis, Cystic Fibrosis Transmembrane Conductance Regulator, Gene Transfer Techniques, Genetic Therapy, Humans