Liposome delivery of the gene for the cystic fibrosis transmembrane conductance regulator data

University of Pittsburgh researchers and collaborators in Britain published results of a Phase I/II trial in 15 adult males with cystic fibrosis. As reported in Nature Medicine, a 20 percent partial restoration of the ion conductance defect was achieved temporarily, with some responses to low chloride in the range for normal values.

The cationic liposomes were delivered by nasal aerosol. Biopsies showed a maximal effect at day 3, which reverted to pretreatment levels after a week. ...