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Article

High-Level Factor VIII Gene Expression <i>In Vivo</i> Achieved by Nonviral Liver-Specific Gene Therapy Vectors

11 Sept 2003

Abstract excerpt

Two liver-specific nonviral gene transfer vectors have been developed to accommodate heterologous genes. The expression cassettes contain (1) a hepatic locus control region from the apolipoprotein E (ApoE) gene (HCR), (2) a liver-specific alpha(1)-antitrypsin promoter (HP), (3) a 1.4-kb truncated factor IX first intron (I) or a synthetic minx intron (mI), (4) a multiple cloning site (MCS) for inserting cDNA...

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PMID: 14503965
DOI: 10.1089/104303403322319381

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