Human Gene Therapy


Human gene therapy is the treatment of human diseases with deoxyribonucleic acid fragments comprising whole genes or parts of genes.

Keywords: gene; disease; viral; nonviral; therapy

Further Reading

Baum C, Dullmann J, Li Z et al. (2003) Side effects of retroviral gene transfer into hematopoietic stem cells. Blood 101: 2099–2114 (Review).

Cavazzana‐Calvo M, Hacein‐Bey S, de Saint Basile G et al. (2000) Gene therapy of human severe combined immunodeficiency (SCID)‐X1 disease. Science 288: 669–672.

Evans JT and Garcia JV (2000) Lentivirus vector mobilization and spread by human immunodeficiency virus. Human Gene Therapy 11: 2331–2339.

Felgner PL (1997) Nonviral strategies for gene therapy. Scientific American 276: 102–106.

Fox JL (2000) Gene‐therapy death prompts broad civil lawsuit. Nature Biotechnology 18: 1136.

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

Friend SH (1999) How DNA microarrays and expression profiling will affect clinical practice. British Medical Journal 319: 1306.

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

Michael A (1996) Financing gene therapy beyond phase II. Gene Therapy 3: 1035–1038.

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

Orford M, Nefedov M, Vadolas J et al. (2000) Engineering EGFP reporter constructs into a 200 kb human globin BAC clone using GET recombination. Nucleic Acids Research 28(84): 1–8.

Penn SG, Rank DR, Hanzel DK and Barker DL (2000) Mining the human genome using microarrays of open reading frames. Nature Genetics 26: 315–318.

Thompson L (2000) Human gene therapy. Harsh lessons, high hopes. FDA Consumer 34(5) [].

Walther W and Stein U (eds) (2000) Gene Therapy of Cancer: Methods and Protocols. Totowa, NJ: Humana Press.

Zhao S (2001) A comprehensive BAC resource. Nucleic Acids Research 29: 141–143.

Contact Editor close
Submit a note to the editor about this article by filling in the form below.

* Required Field

How to Cite close
Ioannou, Panos A(Jan 2006) Human Gene Therapy. In: eLS. John Wiley & Sons Ltd, Chichester. [doi: 10.1038/npg.els.0003841]