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The treatment of hemophilia A: from protein replacement to AAV-mediated gene therapy

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Abstract

Factor VIII (FVIII) is an essential component in blood coagulation, a deficiency of which causes the serious bleeding disorder hemophilia A. Recently, with the development of purification level and recombinant techniques, protein replacement treatment to hemophiliacs is relatively safe and can prolong their life expectancy. However, because of the possibility of unknown contaminants in plasma-derived FVIII and recombinant FVIII, and high cost for hemophiliacs to use these products, gene therapy for hemophilia A is an attractive alternative to protein replacement therapy. Thus far, the adeno-associated virus (AAV) is a promising vector for gene therapy. Further improvement of the virus for clinical application depends on better understanding of the molecular structure and fate of the vector genome. It is likely that hemophilia will be the first genetic disease to be cured by somatic cell gene therapy.

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Acknowledgments

This work was supported by Natural Science Fund of Guangdong.

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Authors and Affiliations

  1. Department of Hematology, The Second Hospital of Shantou University Medical College, 515041, Shantou, China

    Shen Youjin

  2. Department of Laboratory, The Second Hospital of Shantou University Medical College, 515041, Shantou, China

    Yin Jun

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  1. Shen Youjin
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  2. Yin Jun
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Correspondence to Shen Youjin.

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Youjin, S., Jun, Y. The treatment of hemophilia A: from protein replacement to AAV-mediated gene therapy. Biotechnol Lett 31, 321–328 (2009). https://doi.org/10.1007/s10529-008-9869-0

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