Retroviral mediated transfer and expression of human alpha 1-antitrypsin in cultured cells |
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Authors: | F D Ledley H E Grenett D P Bartos S L Woo |
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Institution: | Howard Hughes Medical Institute, Department of Cell Biology, Houston, TX. |
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Abstract: | Genetic deficiency of alpha 1-antitrypsin in man is a predisposing factor to emphysema and a disorder potentially correctable by somatic gene therapy. A full-length human alpha 1-antitrypsin cDNA was cloned into a retroviral vector and introduced into cells which package the recombinant gene in a retroviral capsule. Cells infected with the recombinant retrovirus express human alpha 1-antitrypsin mRNA and protein. The recombinant protein is glycosylated, secreted and exhibits anti-protease activity against human neutrophil elastase. |
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