Future therapies for CF may involve direct replacement or repair of the CFTR gene in the airways of affected patients. This was the focus of several recent CFF RFAs. In order to develop effective therapies, improved vector delivery to the airway epithelium will be required; recent advances in improving airway specificity of viral vectors will be presented. Furthermore, recent technological advances now permit the site-specific repair of genes (such as CFTR) both ex vivo and in vivo; a recent example of in vivo correction of muscular dystrophy will be highlighted. Finally, basal cells play a crucial role in maintaining the airway epithelium; their potential for cellular therapy in CF will be discussed.
Discuss potential gene and cell therapies for CF.
Identify technical challenges for development and delivery of such therapies.
Review how application of such approaches to other diseases may highlight issues relevant to application to CF.