Gene therapy for cystic fibrosis: challenges and future directions.

Abstract

for gene therapy when the gene responsible for the disease was isolated in 1989 (3-5). The wide distribution of possible cellular targets for gene transfer in the CF lung and the absence of a known lung epithelial stem cell suggested that an ex vivo approach to gene therapy would not be feasible (6, 7). Research immediately focused on in vivo approaches for gene transfer that could conveniently be delivered into the airway via aerosols. The ensuing research effort has literally dominated the field ofhuman gene therapy of genetic diseases while establishing the basic principles of in vivo gene delivery. This review summarizes critical scientific issues that have emerged in the development of in vivo gene therapy for CF using adenoviral vectors as a model.