Sustained high-level expression of full-length human factor VIII and restoration of clotting activity in hemophilic mice using a minimal adenovirus vector.

@article{Balagu2000SustainedHE,
  title={Sustained high-level expression of full-length human factor VIII and restoration of clotting activity in hemophilic mice using a minimal adenovirus vector.},
  author={Claudine Balagu{\'e} and Junfang Zhou and Yan-feng Dai and Ramon Alemany and Steven F. Josephs and Grai L. Andreason and Manoj Hariharan and Erum W. Sethi and E. I. Prokopenko Prokopenko and H Christensen Jan and Yan Chun Lou and D Hubert-Leslie and Laura Polledo Ruiz and Wen Wei Zhang},
  journal={Blood},
  year={2000},
  volume={95 3},
  pages={
          820-8
        }
}
The successful prophylactic treatment of hemophilia A by frequent infusions of plasma concentrates or recombinant factor VIII (hFVIII) indicates that gene therapy may be a potential alternative for the treatment of the disease. For efficient delivery and long-term expression of the hFVIII gene, a novel minimal adenovirus (mini-Ad) vector, MiniAdFVIII, has been developed. The vector is devoid of all viral genes and carries the full-length hFVIII cDNA under the control of the human 12.5-kb… CONTINUE READING

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Biochemical, immu BLOOD

  • DD Pittman, EM Alderman, KN Tomkinson, JH Wang, AR Giles, RJ. Kaufman
  • For personal use only. on October
  • 2000

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