State of the Art in Gene Therapy

  • H. J. Haisma
  • M. G. Rots
Conference paper
Part of the Developments in Hematology and Immunology book series (DIHI, volume 38)


The elucidation of the complete human genome with approximately 30.000 different genes will lead to new possibilities for diagnosis and prevention of a wide variety of diseases. In addition, this knowledge may be used for the design of new therapeutics, based on the DNA sequence information. One of these approaches is gene therapy, where new genes are introduced in a patient to correct a specific disease. For many years, gene transfer techniques have been used in the laboratory setting to introduce altered or foreign genes into cells in order to produce a specific, desired outcome. Since the 1990s, gene therapy has been studied as a possible means of modifying the genetic makeup of patients to treat a specific disease.


Cystic Fibrosis Gene Therapy Adenoviral Vector Critical Limb Ischemia Diphtheria Toxin 
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Copyright information

© Springer Science+Business Media Dordrecht 2003

Authors and Affiliations

  • H. J. Haisma
  • M. G. Rots
    • 1
  1. 1.Dept of Therapeutic Gene Modulation, University Centre for PharmacyUniversity of GroningenGroningenThe Netherlands

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