Gp. Kobinger et al., Filovirus-pseudotyped lentiviral vector can efficiently and stably transduce airway epithelia in vivo, NAT BIOTECH, 19(3), 2001, pp. 225-230
Traditional gene therapy vectors have demonstrated limited utility for trea
tment of chronic lung diseases such as cystic fibrosis (CF). Herein we desc
ribe a vector based on a Filovirus envelope protein-pseudotyped HIV vector,
which we chose after systematically evaluating multiple strategies. The ve
ctor efficiently transduces intact airway epithelium from the apical surfac
e, as demonstrated in both in vitro and in vivo model systems. This shows t
he potential of pseudotyping in expanding the utility of lentiviral vectors
. Pseudotyped lentiviral vectors may hold promise for the treatment of CF.