Gene therapy for cystic fibrosis.
The pursuit of an effective genetic therapyfor cysticfibrosis (CF) has led to significant advances in the development of viral and non-viral vector systems. However, obstacles to clinically effective gene transfer in the diseased airways of CF patients remain. Ongoing gene transfer trials in humans are helping to better define those obstacles, some of which relate to host defense and some of which relate to inherent biological properties of the various gene transfer vector systems. In this review, the relevant aspects of airway biology, the pathophysiological CF lung disease, and the characteristics of the currently available vector systems are discussed.
Flotte
T R
TR
University of Florida Genetics Institute, JHMHSC, Gainesville 32610-0266, USA. flottr@peds.ufl.edu
D000256:Adenoviridae / Q000235:genetics / Q000276:immunology; D000402:Airway Obstruction / Q000209:etiology; D000818:Animals; D017321:Clinical Trials, Phase I as Topic; D003550:Cystic Fibrosis / Q000150:complications / Q000235:genetics / Q000473:pathology / Q000628:therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics* / Q000502:physiology; D004274:DNA, Recombinant / Q000008:administration & dosage / Q000627:therapeutic use; D003673:Defective Viruses / Q000235:genetics; D000229:Dependovirus / Q000235:genetics; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000008:administration & dosage / Q000235:genetics / Q000276:immunology / Q000627:therapeutic use; D006801:Humans; D016086:Lentivirus / Q000235:genetics; D008081:Liposomes / Q000008:administration & dosage; D008168:Lung / Q000473:pathology; D014677:Pharmaceutical Vehicles; D011993:Recombinant Fusion Proteins / Q000502:physiology; D012190:Retroviridae / Q000235:genetics
C505032:CFTR protein, human; D004274:DNA, Recombinant; D008081:Liposomes; D014677:Pharmaceutical Vehicles; D011993:Recombinant Fusion Proteins; D019005:Cystic Fibrosis Transmembrane Conductance Regulator