The first demonstration of an AAV vector used in humans was performed in 1995 and involved the delivery of the cystic fibrosis transmembrane regulator (CFTR) gene packaged with the AAV2 capsid (rAAV2-CFTR), into a patient with cystic fibrosis.143 Since this first demonstration, multiple vector designs have been reported. Here, CFTR is linked to cystic fibrosis.