University of North Carolina, Chapel Hill preclinical data

University researchers, using the previously reported mouse model, have determined that certain cells of the airway are more easily induced to take up the gene carried by an adenovirus vector.

The researchers were seeking to understand why low doses of adenovirus vectors carrying CFTR cDNA can correct defective chloride transport in vitro but not in vivo. In their mouse studies, even repeated, high doses of the Ad5-CB adenovirus vector bearing the CF gene failed to restore more than 50 percent of defective transport of chloride, and did not correct the sodium defect at all. ...