Gene therapy for cystic fibrosis: an example for lung gene therapy
- 29 September 2004
- journal article
- review article
- Published by Springer Nature in Gene Therapy
- Vol. 11 (S1) , S43-S50
- https://doi.org/10.1038/sj.gt.3302368
Abstract
Gene therapy is currently being evaluated for a wide range of acute and chronic lung diseases. The requirement of gene transfer into the individual cell types of the complex lung structure will very much depend on the target disease. Over the last decade, the gene therapy community has recognized that there is not even one vector that is good for all applications, but that the gene transfer agent has to be carefully chosen. Gene therapy is particularly attractive for diseases that currently do not have satisfactory treatment options and probably easier for monogenic disorders than for complex diseases. Cystic fibrosis (CF) fulfills these criteria and is therefore a good candidate for gene therapy-based treatment. This review will focus on CF as an example for lung gene therapy and discuss the progress made in this field over the last couple of years.Keywords
This publication has 59 references indexed in Scilit:
- Transfection efficiency and toxicity following delivery of naked plasmid DNA and cationic lipid–DNA complexes to ovine lung segmentsMolecular Therapy, 2003
- Recovery of Airway Cystic Fibrosis Transmembrane Conductance Regulator Function in Mice with Cystic Fibrosis After Single-Dose Lentivirus-Mediated Gene TransferHuman Gene Therapy, 2002
- Uptake of plasmid/glycosylated polymer complexes and gene transfer efficiency in differentiated airway epithelial cellsThe Journal of Gene Medicine, 2002
- An algorithm for finding protein–DNA binding sites with applications to chromatin- immunoprecipitation microarray experimentsNature Biotechnology, 2002
- Functional Evidence of CFTR Gene Transfer in Nasal Epithelium of Cystic Fibrosis Mice in Vivo Following Luminal Application of DNA Complexes Targeted to the Serpin-Enzyme Complex ReceptorMolecular Therapy, 2002
- Targeting Transgene Expression for Cystic Fibrosis Gene TherapyMolecular Therapy, 2001
- High and Sustained Transgene Expression in Vivo from Plasmid Vectors Containing a Hybrid Ubiquitin PromoterMolecular Therapy, 2001
- Gene Therapy for Cystic Fibrosis with Aerosolized Adenovirus-CFTR: Characterization of the Aerosol and Scintigraphic Determination of Lung Deposition in BaboonsJournal of Aerosol Medicine, 2001
- Control of Cystic Fibrosis Transmembrane Conductance Regulator Expression by BAP31Published by Elsevier ,2001
- Role of Alveolar Macrophages in Rapid Elimination of Adenovirus Vectors Administered to the Epithelial Surface of the Respiratory TractHuman Gene Therapy, 1997