Adeno-associated virus data

In rabbits, company scientists and colleagues transferred the gene for CFTR by bronchoscope to a single lung lobe, and achieved stable expression of the protein for up to six months specifically in the infected lobe and not the other half of the lung.

As detailed in the Proceedings of the National Academy of Sciences, the AAV vector produced a CFTR protein that is truncated at its amino terminal and modified with a fusion peptide. This modification served as a tag to detect the gene. The company will probably use the naturally occurring CFTR in human trials, which could start by the end of 1994. Toxicity testing in primates is underway. ...