GENE THERAPY

  • Irena Milisav Laboratorij za nevroendokrinologijo – molekularna celična fiziologija Inštitut za patološko fiziologijo Medicinska fakulteta Zaloška 4 1000 Ljubljana
Keywords: gene therapy, clinical studies, antisense therapy, cancer

Abstract

Background. Gene therapy is a novel approach to treat, cure, or ultimately prevent disease by changing the expression of person’s genes.

Gene therapy is in its inflancy, and current gene therapy is primarily experimental, with most human clinical trials only in the research stages. There were 600 studies initiated from 1990–2001, mostly in USA.

Conclusions. Most of the gene therapy protocols currently tested are for treating various cancers. This is followed by the treatment of cardiovascular diseases, genetic diseases and infectious diseases, the latter mostly to control the HIV infection. About 5 studies devoted to cancer treatment are in the third phase of the trials and may be approved for clinical use in the near future.

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References

Anon. Guidance for industry. Guidance for human somatic cell therapy and gene therapy. US Dept. of Health and Human Services, FDA, Center for Biologics Evaluation and Research, 1998: 3–4.

Clinical trials in human gene transfer. http://www4.od.nih.gov/oba/rac/ clinicaltrial.htm, Accessed: August 2002.

Romano G, Pacilio C, Giordano A. Gene transfer technology in therapy: Current applications and future goals. The Oncologist 1998; 3: 255–63.

Koenig S. Antibody gene therapy with adeno-associated viral vectors. Curr Opp Mol Ther 2001; 3: 433–3.

FDA. http://www.fda.gov/cber/gene.htm, Accessed: September 2001.

Pachori AS, Huentelman MJ, Francis SC, Gelband CH, Katovich MJ, Raizada MK. The future of hypertension therapy: Sense, antisense or nonsense. Hypertension 2001; 37: 357–64.

FDA. http://www.fda.gov/, Accessed: September 2002.

Mountain A. Gene therapy: the first decade. TIBTECH 2000; 18: 119–26.

Blaese RM, Culver KW, Anderson WF. The ADA human gene therapy clinical protocol. Hum Gene Ther 1990; 1: 331–7.

The Jeffrey Modell Foundation. Severe combined immunodeficiency. http:/ /www.jmfworld.org.html/severe_combined_immunodeficien.html, Accessed: May 2002.

OMIM. http://www.ncbi.nlm.nih.gov/htbin-post/Omim/dispmim?202500, Accessed: May 2002.

French-Anderson W. Gene therapy: The best of times, the worst of times. Science 2000; 288: 627–9.

Blaese MR, Culver KW, Miller AD et al. T lymphocyte-directed gene therapy for ADA- SCID: Initial trial results after 4 years. Science 1995; 270: 475–80.

Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, Gross F et al. Gene therapy of human severe combined immunodeficiency (SCID)-XI disease. Science 2000; 288: 669–9.

J. Gene Medicine. http://www.wiley.co.uk/genetherapy, Accessed: September 2001.

Lehrman S. Virus treatment questioned after gene therapy death. Nature 1999; 401: 517–8.

Smaglik P. Clinical trials at gene therapy institute. Nature 2000; 405: 497–7.

Wadman M. NIH under fire over gene-therapy trials. Nature 2000; 403: 237–7.

Smith AE. Gene therapy – where are we? Lancet 1999; 354: Suppl I: 1–4.

Marshall E. Panel reviews risks of germ line changes. Science 2001; 294: 2268–9.

Zivin JA. Understanding clinical trials. Sci Am 2000; April: 49–55.

Marshall E. Gene Gemisch cures Sicle cell in mice. Science 2001; 294: 2268–8.

Hunt S. Technology evaluation: MetXia-P450, Oxford BioMedica. Curr Opp Mol Ther 2001; 3: 595–8.

Check E. A tragic setback. Nature 2002; 420: 116–8.

How to Cite
1.
Milisav I. GENE THERAPY. TEST ZdravVestn [Internet]. 1 [cited 5Aug.2024];72(2). Available from: http://vestnik-dev.szd.si/index.php/ZdravVest/article/view/1796
Section
Review