Gene therapy for cystic fibrosis: a potential cure

Abstract

Cystic fibrosis is a lethal autosomal recessive disorder. In 1989, the cystic fibrosis gene was isolated on chromosome 7. Positional cloning, linkage analysis, saturation cloning, and chromosome jumping enabled the isolation and cloning of the gene that encodes the cystic fibrosis transmembrane conductance regulator. Current work focuses on the safe, efficient delivery of a functional cystic fibrosis transmembrane conductance gene to patients who are afflicted with cystic fibrosis and the expression of the gene in somatic cells to correct the genetic defect and restore chloride channel function. It is hoped that successful gene therapy will be a feasible and cost-efficient approach that will lead to a cure.