Gene therapy for cystic fibrosis: a clinical perspective.
Since the isolation of the cystic fibrosis (CF) gene in 1989, the potential for gene therapy for this disease has existed. Although current treatments have resulted in a mean life expectancy of approximately 30 years, there is clearly a need for more effective therapy. A large number of studies have now assessed both in vitro and in vivo CFTR gene transfer into cell lines, animal models and most recently in CF subjects. Despite this rapid progress several difficulties remain including efficient in vivo gene transfer and the measurement of end-points to assess such gene transfer. This article reviews some of the clinically related aspects of gene therapy for CF.
Alton
E W
EW
Royal Brompton Hospital, National Heart and Lung Institute, London, UK.
Geddes
D M
DM
D000818:Animals; D002712:Chlorides / Q000378:metabolism; D003550:Cystic Fibrosis / Q000235:genetics / Q000628:therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator; D018076:DNA, Complementary / Q000008:administration & dosage / Q000235:genetics; D004195:Disease Models, Animal; D004311:Double-Blind Method; D005814:Genes, Viral; D015316:Genetic Therapy / Q000379:methods; D005822:Genetic Vectors / Q000008:administration & dosage; D006801:Humans; D008081:Liposomes; D008168:Lung / Q000378:metabolism; D008565:Membrane Proteins / Q000096:biosynthesis / Q000235:genetics; D051379:Mice; D008822:Mice, Transgenic; D009330:Nebulizers and Vaporizers; D009928:Organ Specificity; D011401:Promoter Regions, Genetic; D011993:Recombinant Fusion Proteins / Q000096:biosynthesis; D016896:Treatment Outcome
C505032:CFTR protein, human; D002712:Chlorides; D018076:DNA, Complementary; D008081:Liposomes; D008565:Membrane Proteins; D011993:Recombinant Fusion Proteins; D019005:Cystic Fibrosis Transmembrane Conductance Regulator