Gene therapy for cystic fibrosis
Flotte, T.R.
Current Opinion in Molecular Therapeutics 1(4): 510-516
1999
ISSN/ISBN: 1464-8431 PMID: 11713767 Document Number: 505436
The pursuit of an effective genetic therapyfor cysticfibrosis (CF) has led to significant advances in the development of viral and non-viral vector systems. However, obstacles to clinically effective gene transfer in the diseased airways of CF patients remain. Ongoing gene transfer trials in humans are helping to better define those obstacles, some of which relate to host defense and some of which relate to inherent biological properties of the various gene transfer vector systems. In this review, the relevant aspects of airway biology, the pathophysiological CF lung disease, and the characteristics of the currently available vector systems are discussed.