Gene therapy progress and prospects: cystic fibrosis.
Since the cloning of the cystic fibrosis gene (CFTR) in 1989, 18 clinical trials have been carried out, including five in the 2 years reviewed here. Most trials demonstrated proof-of-principle for gene transfer to the airway. However, gene transfer efficiency with each of the three gene transfer agents (adenovirus (Ad), adeno-associated virus 2 (AAV2) and cationic liposomes) was low, and most likely insufficient to achieve clinical benefit. Here, we will review the clinical and pre-clinical progress for the last 2 years (2000-2001) and briefly speculate on future prospects for the next 2 in CF gene therapy.
D000818:Animals; D017321:Clinical Trials, Phase I as Topic; D003550:Cystic Fibrosis / Q000235:genetics / Q000628:therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics; D004847:Epithelial Cells / Q000378:metabolism; D015870:Gene Expression; D018390:Gene Targeting; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000008:administration & dosage; D006801:Humans; D008168:Lung; D016376:Oligonucleotides, Antisense / Q000008:administration & dosage
C505032:CFTR protein, human; D016376:Oligonucleotides, Antisense; D019005:Cystic Fibrosis Transmembrane Conductance Regulator