AAV Vector-Mediated Liver Gene Therapy and Its Implementation for Hemophilia

  • Hiroaki Mizukami
  • Jun Mimuro
  • Tsukasa Ohmori
  • Yoichi Sakata
  • Keiya Ozawa


Adeno-associated virus (AAV) vector is widely used in gene transfer purposes. Not only the experimental gene transfer but also applications toward disease therapy are promising. Numerous attempts are ongoing for this purpose. One of the most prominent examples is the sustained clinical benefit in hemophilia gene therapy targeting the liver. These successes have been brought chiefly by the progress in vectorology, especially capsid development. At present, one of the biggest issues is the presence of neutralizing antibody (NAb) against AAV vector capsid. Challenges have been made to conquer this problem. Although there are still some hurdles for wide clinical application, use of this vector will soon be a common practice for the treatment of suitable disease conditions.


AAV vector Serotype Hemophilia Neutralizing antibody Empty capsid 


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Copyright information

© Springer Japan 2016

Authors and Affiliations

  • Hiroaki Mizukami
    • 1
  • Jun Mimuro
    • 2
    • 3
  • Tsukasa Ohmori
    • 2
  • Yoichi Sakata
    • 2
  • Keiya Ozawa
    • 1
    • 4
  1. 1.Division of Genetic Therapeutics, Center for Molecular MedicineJichi Medical UniversityShimotsukeJapan
  2. 2.Division of Cell and Molecular Medicine, Center for Molecular MedicineJichi Medical UniversityShimotsukeJapan
  3. 3.Department of Internal MedicineIshibashi General HospitalShimotsukeJapan
  4. 4.IMSUT Hospital, The Institute of Medical ScienceThe University of TokyoTokyoJapan

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