Article
A novel SV40-based vector successfully transduces and expresses an alpha 1-antitrypsin ribozyme in a human hepatoma-derived cell line.
Gene therapy - 1 Jan 1999
Zern M A, Ozaki I, Duan L, Pomerantz R, Liu S L, Strayer D S
Abstract excerpt
Alpha 1-antitrypsin (alpha 1AT) deficiency disease is one of the more common hereditary disorders that affects the liver and lung. The liver disease of alpha 1AT deficiency is generally thought to be caused by the accumulation of an abnormal alpha 1AT protein in hepatocytes, whereas the lung disease is thought to be due to a relative lack of the normal protein in the circulation. Therefore, one possible approach...
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