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Rare Diseases pp 131-157 | Cite as

Adeno-Associated Virus Gene Therapy and Its Application to the Prevention and Personalised Treatment of Rare Diseases

  • Konstantina GrosiosEmail author
  • Harald Petry
  • Jacek Lubelski
Chapter
  • 962 Downloads
Part of the Advances in Predictive, Preventive and Personalised Medicine book series (APPPM, volume 6)

Abstract

Gene therapy has always found a home in rare diseases and the approval of Glybera® for lipoprotein lipase deficiency has marked a major milestone in gene therapy and has brought this type of therapy even closer to clinical practice. There is little closer to the personalisation of medicine than the ability to repair or restore the function of a person’s own faulty genes, the core principle of gene therapy. It is therefore reasonable to argue that gene therapy is the ultimate personalised medicine. At the same time, advances in scientific understanding and technological ability to analyse the human genome imply that gene therapy could also be used to prevent the development of disease. Using examples from the AAV-based gene therapy field as is applied to rare diseases, the first section of this chapter aims to illustrate how gene therapy aligns to the principles of PPPM whereas the second part of the chapter intends to provide an in depth review of the developments in the AAV field that underpin the use of these viruses as gene therapy delivery systems.

PPPM-Related Keywords

Gene therapy Adeno-associated viral vectors Rare disease prevention Rare disease personalised treatment Alipogene tiparvovec Glybera® Predictive, preventive and personalised medicine 

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Copyright information

© Springer Science+Business Media Dordrecht 2015

Authors and Affiliations

  • Konstantina Grosios
    • 1
    Email author
  • Harald Petry
    • 1
  • Jacek Lubelski
    • 1
  1. 1.uniQure B.VAmsterdamThe Netherlands

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