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Gene transfer vectors as medicinal products: risks and benefits

Part 1. Genetics

1.7. Gene Therapy

Short Specialist Review

  1. Christian J. Buchholz,
  2. Klaus Cichutek

Published Online: 15 JUL 2005

DOI: 10.1002/047001153X.g107304

Encyclopedia of Genetics, Genomics, Proteomics and Bioinformatics

Encyclopedia of Genetics, Genomics, Proteomics and Bioinformatics

How to Cite

Buchholz, C. J. and Cichutek, K. 2005. Gene transfer vectors as medicinal products: risks and benefits. Encyclopedia of Genetics, Genomics, Proteomics and Bioinformatics. 1:1.7:100.

Author Information

  1. Paul-Ehrlich-Institut, Langen, Germany

Publication History

  1. Published Online: 15 JUL 2005

Abstract

Gene transfer medicinal products (GT-MPs) include a broad range of product types as for example, genetically modified somatic cells, viral and nonviral vectors, or carrier-free nucleic acids used in humans for preventive, therapeutic, or in vivo diagnostic purposes. Treatment is targeted not only at inherited diseases but also at common disorders such as cancer, cardio-vascular, and infectious diseases. About a thousand clinical trails using GT-MPs are ongoing or have been completed worldwide, mainly performed in the United States and in the European Union. Up to now, however, only one GT-MP has been licensed, licensing applications in the European Union are expected starting from 2005. In the European Union, marketing authorization for GT-MPs is obtained following the centralized procedure via the European Medicines Evalution Agency (EMEA). Coordinated by the EMEA, experts from national authorities are reviewing applications and give scientific advice to pharmaceutical industry developing medicinal products. This article summarizes the various concepts and definitions of GT-MPs, their state of development, clinical approaches, serious adverse events, and relevant regulatory documents and activities.

Keywords:

  • gene therapy;
  • vector systems;
  • clinical trials;
  • regulation;
  • marketing authorization