Abstract
Cystic fibrosis (CF) is the most common recessive lethal inherited disorder among Caucasian populations. The genetic basis of cystic fibrosis was resolved in 1989 when the CFTR (cystic fibrosis transmembrane conductance regulator) gene was cloned. Until recently, few CF patients survived childhood and other than transplantation, there is no effective therapy for CF lung disease. The realisation of gene therapy represents the best hope for these patients.
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