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Vectors and Gene Therapy

Pharmacology

  1. Edward A. Burton1,
  2. David J. Fink2,
  3. Joseph C. Glorioso2

Published Online: 15 SEP 2006

DOI: 10.1002/3527600906.mcb.200400087

Reviews in Cell Biology and Molecular Medicine

Reviews in Cell Biology and Molecular Medicine

How to Cite

Burton, E. A., Fink, D. J. and Glorioso, J. C. 2006. Vectors and Gene Therapy. Reviews in Cell Biology and Molecular Medicine. .

Author Information

  1. 1

    University of Pittsburgh, Pittsburgh, PA, USA

  2. 2

    University of Michigan, Ann Arbor, MI, USA

Publication History

  1. Published Online: 15 SEP 2006

Abstract

Gene therapy may be defined as the introduction of genetic sequences into cells of a living organism, resulting in an alteration of cell or tissue function in a manner that corrects or treats a pathological process.

This might be accomplished in one of two broad ways: (1) The therapeutic genetic material is transported ex vivo into cells taken from a patient—the cells can be subsequently reintroduced, with or without expansion, as an homologous cell transplant. The reintroduced cells engraft and produce a therapeutic product that can alter the biology of the transduced cell to correct a genetic abnormality or deficiency, or may serve as a factory for production of a protein product used by other tissues exposed to the circulation. (2) The therapeutic genetic material is introduced into the cells of the body by direct in vivo gene transfer. Direct in vivo gene transfer may be used if procurement of appropriate cell explants requires unacceptably invasive procedures, or for gene transfer to essentially nonregenerating tissues (e.g. brain or heart).

The genetic material transferred (“transgene”) may encode a complete therapeutic gene to replace a defective function, or coding sequences whose products interfere with an acquired or inherited pathologic cellular function. Gene transfer may also be used to enhance normal function to achieve a therapeutic effect, such as the induction of an immune response or the reversal of autoimmune activity. Finally, a large effort in gene therapy has been directed toward the use of gene transfer to destroy unwanted tissue, such as malignant cells, or fight infectious agents, such as human immune deficiency virus (HIV).

The diverse potential gene therapy applications suggest that genes as molecular medicines hold great promise for the future. In part this promise is beginning to be realized, but much more research (and testing in patients) is required before gene therapy becomes standard medical practice.

Keywords:

  • Adeno-associated Virus;
  • Adenovirus;
  • Ex vivo Gene Transfer;
  • Gene Therapy;
  • Gene Transfer Vector;
  • Herpes Simplex Virus;
  • In vivo Gene Transfer;
  • Lentivirus;
  • Nonviral Vector;
  • Replication defective;
  • Tropism;
  • Viral Vector