Cystic fibrosis gene therapies being tested

“Crippled” adenovirus selected (non-integrating, replication defective, respiratory virus) – Gene therapy trials – 3 Research teams, 10 patients/team. 2 teams administered virus via aerosol delivery into nasal passages ad lungs. 1 team administered virus via nasal passages only. Only transient expression observed →because adenovirus does not integrate into genome like retroviruses