Preclinical evaluation of a recombinant adeno-associated virus vector expressing human alpha-1 antitrypsin made using a recombinant herpes simplex virus production method.

@article{Chulay2011PreclinicalEO,
  title={Preclinical evaluation of a recombinant adeno-associated virus vector expressing human alpha-1 antitrypsin made using a recombinant herpes simplex virus production method.},
  author={JeffreyD. Chulay and Guo-jie Ye and Darby L. Thomas and David R. Knop and Janet M. Benson and Julie A. Hutt and Gensheng Wang and Margaret R Humphries and Terence R. Flotte},
  journal={Human gene therapy},
  year={2011},
  volume={22 2},
  pages={
          155-65
        }
}
Recombinant adeno-associated virus (rAAV) vectors offer promise for gene therapy of alpha-1 antitrypsin (AAT) deficiency. A toxicology study in mice evaluated intramuscular injection of an rAAV vector expressing human AAT (rAAV-CB-hAAT) produced using a herpes simplex virus (HSV) complementation system or a plasmid transfection (TFX) method at doses of 3 × 10(11) vg (1.2 × 10(13) vg/kg) for both vectors and 2 × 10(12) vg (8 × 10(13) vg/kg) for the HSV-produced vector. The HSV-produced vector… CONTINUE READING

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