Gene Therapy


Gene therapy can be defined as the deliberate transfer of genetic material for a therapeutic purpose.

Keywords: gene therapy; cancer; germ line; transcription; ethics

Further Reading

Bolognani F and Goya RG (2001) Gene therapy in the neuroendocrine system. Neuroendocrinology 73: 75–83.

Chuah MK, Collen D and Vandendriessche T (2001) Gene therapy for haemophilia. Journal of Gene Medicine 3: 3–20.

Jenks S (2000) Gene therapy death – everyone has to share in the guilt. Journal of the National Cancer Institute 92: 98–100.

Kresina T (ed.) (2001) An Introduction to Molecular Medicine and Gene Therapy New York, NY: Wiley‐Liss.

Morgan RA (1999) Genetic strategies to inhibit HIV. Molecular Medicine Today 5: 454–458.

Morgan RA and Blaese RM (1999) Gene therapy: lessons learnt from the last decade. British Medical Journal 319: 1310.

Pouton CW and Seymour LW (2001) Key issues in non viral gene delivery. Advanced Drug Delivery Reviews 46: 187–203.

Robinson HL (1999) DNA vaccines: basic mechanism and immune responses. International Journal of Molecular Medicine 4: 549–555.

Stephenson J (2000) Gene therapy trial shows clinical efficacy. Journal of the American Medical Association 283: 589–590.

Zanjani ED and Anderson WF (1999) Prospects for in utero human gene therapy. Science 285: 2084–2088.

Web Links

Adenosine deaminase (ADA); MIM number: 102700. OMIM:‐post/Omim/dispmim?102700

Adenosine deaminase (ADA); LocusID: 100. LocusLink:

Cystic fibrosis transmembrane conductance regulator (CFTR); MIM number: 602421. OMIM:‐post/Omim/dispmim?602421

Cystic fibrosis transmembrane conductance regulator (CFTR); LocusID: 1080. LocusLink:

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How to Cite close
Sikora, Karol(Jan 2006) Gene Therapy. In: eLS. John Wiley & Sons Ltd, Chichester. [doi: 10.1038/npg.els.0005835]