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Therapeutic Serum Concentrations of Human Alpha-1-antitrypsin After Adenoviral-mediated Gene Transfer Into Mouse Hepatocytes by Mark A. Kay; Frank Graham; Frances Leland; Savio L. C. Woo is a Medicine article available to read on EtoBox.

What is Therapeutic Serum Concentrations of Human Alpha-1-antitrypsin After Adenoviral-mediated Gene Transfer Into Mouse Hepatocytes about?

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 uiuo and direct in uiuo transduction of hepatocytes in dogs and rodents. This approach led to low levels of the human protein in the serum of recipients. In this study, recombinant adenoviral vectors that express the human alpha-1-antitrypsin cDNA under the transcriptional control of the phosphoglycerate kinase (PGK) or RSV-LTR promoters have been constructed and used for the direct transduction of mouse hepatocytes in uiuo. The animals transduced with the recombinant adenoviral vectors had therapeutic serum levels of human alpha-1-antitrypsin of up to 700 pg/mL. Thus, adenovirus-mediated gene transfer of the hAAT cDNA into the liver was able to produce therapeutic serum concentrations of protein. (HEPATOLOGY 1995;21:815-819.) Alpha-1-antitrypsin (AAT) deficiency results in early chronic obstructive pulmonary emphysema or infantile liver chirrosis. AA

Who reads Therapeutic Serum Concentrations of Human Alpha-1-antitrypsin After Adenoviral-mediated Gene Transfer Into Mouse Hepatocytes?

Author

Mark A. Kay; Frank Graham; Frances Leland; Savio L. C. Woo

Publisher

Ovid Technologies (Wolters Kluwer Health)

Published

1995

Language

EN

Field

Medicine (Health Sciences)