Gene Therapy: Technology


Gene therapy promises to be the next revolution in medicine, in which drugs made of genetic material can be used to treat disease. Although success has been limited thus far, new technological advances are providing hope for the future.

Keywords: gene; virus; nonviral vector; gene delivery

Figure 1.

Gene therapy attempts to correct disease by introducing genetic material into cells. A dysfunctional gene can be corrected by the introduction of a normal copy of the gene (top panel). Many different diseases, both genetic and acquired, can be targets for gene therapy (middle panel). Viruses can be used as agents to transfer genetic material into human cells (bottom panel). CF: cystic fibrosis; HIV: human immunodeficiency virus.

Figure 2.

Two different methods can be used to introduce genetic material into the body. Ex vivo therapy involves the removal of cells from the patient and transfer of the gene into the cells outside of the body. The cells are then returned to the patient to correct disease. For in vivo gene transfer, the vector carrying the gene is directly injected into the body. Once in the body, the vector must transfer the genetic material to the appropriate cells in order to correct disease.


Further Reading

Friedmann T (1997) Overcoming the obstacles to gene therapy. Scientific American 276: 96–101.

Freidmann T (ed.) (1999) The Development of Human Gene Therapy. Cold Spring Harbor, NY: Cold Spring Harbor Laboratory Press.

Kay MA, Glorioso JC and Naldini L (2001) Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics. Nature Medicine 7: 33–40.

Schatzlein AG (2001) Non‐viral vectors in cancer gene therapy: principles and progress. Anticancer Drugs 12: 275–304.

Somia N and Verma IM (2000) Gene therapy: trials and tribulations. Nature Reviews Genetics 1: 91–99.

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Haberman, Rebecca P, and Samulski, R Jude(Jan 2006) Gene Therapy: Technology. In: eLS. John Wiley & Sons Ltd, Chichester. [doi: 10.1038/npg.els.0005836]