Gene transfer to the lung: lessons learned from more than 2 decades of CF gene therapy.
Gene therapy is currently being developed for a wide range of acute and chronic lung diseases. The target cells, and to a degree the extra and intra-cellular barriers, are disease-specific and over the past decade the gene therapy community has recognized that no one vector is good for all applications, but that the gene transfer agent (GTA) has to be carefully matched to the specific disease target. 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) fulfils 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, but lessons learned may be applicable to other target diseases.
Griesenbach
Uta
U
Department of Gene Therapy, Faculty of Medicine at the National Heart and Lung Institute, Imperial College London, Manresa Road, London SW36LR, UK. u.griesenbach@imperial.ac.uk
Alton
Eric W F W
EW
D000818:Animals; D002986:Clinical Trials as Topic; D003550:Cystic Fibrosis / Q000628:therapy*; D005100:Expectorants / Q000008:administration & dosage / Q000627:therapeutic use; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D009093:Mucus / Q000737:chemistry / Q000378:metabolism; D020545:Respiratory Mucosa / Q000378:metabolism
D005100:Expectorants