PROSPECTS FOR GENE-THERAPY FOR CYSTIC-FIBROSIS

Despite advances in conventional treatments for cystic fibrosis (CF), the disease is still associated with significant morbidity and mortali ty, The cloning of the cystic fibrosis transmembrane conductance regul ator (CFTR) gene and the understanding of the functions of the CFTR pr otein have led to the development of novel treatment strategies, inclu ding gene therapy. Here, we review the underlying molecular defect in CF cells, and the progress in gene-transfer studies from in vitro work through to clinical trials. We discuss the problems encountered, the end points used to assess efficacy, and the likely future directions o f the field.