Is DNA destiny? A cure for cystic fibrosis.
Cystic fibrosis (CF) remains an attractive target for cure by gene therapy. Results from several trials are reviewed in this article and have shown that mature airway epithelial cells are relatively resistant to gene transfer, that host immune responses determine the duration of transgene expression and define the toxicity, and that the efficiency of transfection remains low. Significant hurdles to the development of gene therapy remain, including the definition of efficacy endpoints, the ability to produce enough material, and the ability to dose the entire lung. Nonetheless, invaluable insights into CF and pulmonary biology have been gained in the gene therapy research effort.
Department of Clinical Pharmacology, University of Pennsylvania, Philadelphia, USA.
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Robinson
D000260:Adenoviruses, Human; D002986:Clinical Trials as Topic; D003550:Cystic Fibrosis / Q000628:therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000502:physiology; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans
C505032:CFTR protein, human; D019005:Cystic Fibrosis Transmembrane Conductance Regulator