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somatic nonreproduc-tive; not an egg or sperm in vitro "in glass"; in lab apparatus, rather than within a living organism

One day, gene therapy may also be used to treat a variety of hereditary and nonhereditary diseases, ranging from loss-of-function disorders such as muscular dystrophy and hemophilia, to gain-of-function disorders such as sickle cell disease, to viral diseases such as HIV-AIDS. Active areas of research include improvements in the methods of gene delivery to the individual tissues and cells of the body and the modulation of the immune response to gene delivery. Many challenges remain to the successful maturation of gene therapy from the laboratory to the clinical setting. see also Cancer; Cystic Fibrosis; Disease, Genetics of; Embryonic Stem Cells; Gene Discovery; Gene Therapy: Ethical Issues; Hemophilia; Muscular Dystrophy; Retrovirus; Ribozyme; Severe Combined Immune Deficiency; Virus.

Michael A. Hauser


Beardsley, T. "Working under Pressure." Scientific American 282 (2000): 34.

Clark, William R. The New Healers: The Promise and Problems of Molecular Medicine in the Twenty-first Century. New York: Oxford University Press, 1999.

Vogel, G. "Gene Therapy: FDA Moves against Penn Scientist." Science 290 (2000): 2049-2051.

Internet Resource

Institute for Human Gene Therapy. <>.

Gene Therapy: Ethical Issues

Gene therapy introduces or alters genetic material to compensate for a genetic mistake that causes disease. It is hoped that gene therapy can treat or cure diseases for which no other effective treatments are available. However, many unique technical and ethical considerations have been raised by this new form of treatment, and several levels of regulatory committees have been established to review each gene therapy clinical trial prior to its initiation in human subjects. Ethical considerations include deciding which cells should be used, how gene therapy can be safely tested and evaluated in humans, what components are necessary for informed consent, and which diseases and/or traits are eligible for gene therapy research.

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