Gene delivery systems - Gene therapy vectors for cystic fibrosis
Journal of Cystic Fibrosis , Volume 3 - Issue SUPPL. 2 p. 203- 212
Gene delivery systems (GDS) play a central role in the development of gene therapy strategies for Cystic Fibrosis (CF). Further, these systems are important tools in studies with cultured cells and in animal models. In this review, we describe the properties of several viral and synthetic gene delivery systems, and evaluate their possible application in gene therapy of CF. While many gene delivery systems give satisfactory results in cultured or animal studies, none of these systems has been shown to fulfil all the requirements of safety and efficacy for use in CF patients. The intact airway epithelium, the most important target in CF gene therapy, proves to be well protected against invading vector systems.
|Cystic fibrosis transmembrane conductance regulator, Gene therapy/ethics/methods, Gene transfer techniques, Genetic vectors, Lung|
|Journal of Cystic Fibrosis|
|Organisation||Biophysical Genomics, Department Cell Biology & Genetics|
Klink, D, Schindelhauer, D, Laner, A, Tucker, T, Bebok, Z, Schwiebert, E.M, … Scholte, B.J. (2004). Gene delivery systems - Gene therapy vectors for cystic fibrosis. Journal of Cystic Fibrosis (Vol. 3, pp. 203–212). doi:10.1016/j.jcf.2004.05.042