Epstein–Barr Virus/Human Vector Provides High-Level, Long-Term Expression of α1-Antitrypsin in Mice

  title={Epstein–Barr Virus/Human Vector Provides High-Level, Long-Term Expression of $\alpha$1-Antitrypsin in Mice},
  author={Stephanie M Stoll and Christopher R. Sclimenti and Edward J. Baba and Leonard Meuse and Mark A. Kay and Mich{\'e}le P. Calos},
  journal={Molecular Therapy},
We have constructed plasmid DNA vectors that contain Epstein-Barr virus (EBV) sequences and the human gene (SERPINA1) encoding alpha1-Antitrypsin (AAT). We demonstrate that a plasmid carrying the full SERPINA1 on a 19-kb genomic fragment and the EBV gene EBNA1 and its family of repeats binding sites undergoes efficient extrachromosomal replication in dividing mammalian tissue culture cells. Therefore, use of a whole genomic therapeutic gene to provide both replication and gene expression may be… 

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