Cystic Fibrosis Research Today is a free monthly online journal that collates and summarizes the latest research about Cystic Fibrosis, including details on symptoms, genetics, treatment, information.

Non-viral vectors in cystic fibrosis gene therapy: progress and challenges.

Montier T, Delépine P, Pichon C, Férec C, Porteous DJ, Midoux P

Unité INSERM 613, Université de Bretagne Occidentale, Institut de Synergie des Sciences et de la Santé, avenue Foch, 29609 Brest cedex, France. tristan.montier@univ-brest.fr

Although the viability of cystic fibrosis (CF) gene transfer to airway epithelium has been demonstrated in vitro and in animal models, so far none of the clinical investigations using adenovirus, adeno-associated virus, lentivirus, cationic lipids or polymers has shown a persistent correction of the ion transport defects that occur in CF. Despite disappointing results, these studies have shown that non-viral vectors could represent a viable alternative for gene therapy in CF airway epithelium. The transfer efficiency of non-viral vectors is currently low, however, and thus these systems are not clinically relevant as yet. Before clinical application, several limitations encountered by non-viral delivery systems must be addressed. Recent progress has been made towards overcoming these limitations and towards making non-viral gene therapy a more realistic option for CF.

Published 19 October 2004 in Trends Biotechnol, 22(11): 586-92.
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