Therapeutic serum concentrations of human alpha‐1‐antitrypsin after adenoviral‐mediated gene transfer into mouse hepatocytes
Mark A. Kay, Frank Graham, Frances Leland, Savio L. C. Woo – 1 March 1995 – Alpha‐1‐antitrypsin is a relatively common genetic deficiency that results in early emphysema. The liver as the natural source of most alpha‐1‐antitrypsin synthesis was the target organ selected for gene replacement therapy studies. Previous work used recombinant retroviral vectors that encode the human alpha‐1‐antitrypsin cDNA for ex vivo and direct in vivo transduction of hepatocytes in dogs and rodents. This approach led to low levels of the human protein in the serum of recipients.