Gene Therapy

gene therapy, the use of genes and the techniques of genetic engineering in the treatment of a genetic disorder or chronic disease. There are many techniques of gene therapy, all of them still in experimental stages. The two basic methods are called in vivo and ex vivo gene therapy. The in vivo method inserts genetically altered genes directly into the patient; the ex vivo method removes tissue from the patient, extracts the cells in question, and genetically alters them before returning them to the patient.

The challenge of gene therapy lies in development of a means to deliver the genetic material into the nuclei of the appropriate cells, so that it will be reproduced in the normal course of cell division and have a lasting effect. One technique involves removing cells from a patient, fortifying them with healthy copies of the defective gene, and reinjecting them into the patient. Another involves inserting a gene into an inactivated or nonvirulent virus and using the virus's infective capabilities to carry the desired gene into the patient's cells. A liposome, a tiny fat-encased pouch that can traverse cell membranes, is also sometimes used to transport a gene into a body cell. Another approach employing liposomes, called chimeraplasty, involves the insertion of manufactured nucleic acid molecules (chimeraplasts) instead of entire genes to correct disease-causing gene mutations. Once inserted, the gene may produce an essential chemical that the patient's body cannot, remove or render harmless a substance or gene causing disease, or expose certain cells, especially cancerous cells, to attack by conventional drugs.

Gene therapy was first used in humans in 1990 to treat a child with adenosine deaminase deficiency, a rare hereditary immune disorder (see immunity). Gene therapy has since been used experimentally to treat a number of conditions, including advanced metastatic melanoma, a myeloid disorder, and a rare hereditary condition that leads to severely impaired vision. Despite the hope that gene therapy can be used to treat cancer, genetic diseases, and AIDS, there are concerns that the immune system may attack cells treated by gene therapy, that the viral vectors could mutate and become virulent, or that altered genes might be passed to succeeding generations. In a few instances trials have been halted when a patient has died or developed disease after undergoing gene therapy.

In the United States, gene therapy techniques must be approved by the federal government. The Recombinant DNA Advisory Committee of the National Institutes of Health oversees gene therapy experiments. Like drugs, products must pass the requirements of the Food and Drug Administration. Gene therapy is a competitive and potentially lucrative field, and patents have been awarded for certain techniques.

See J. Lyon and P. Gorner, Altered Fates: Gene Therapy and the Retooling of Human Life (1995).

The Columbia Encyclopedia, 6th ed. Copyright© 2014, The Columbia University Press.

Gene Therapy: Selected full-text books and articles

Issues and Dilemmas of Biotechnology: A Reference Guide
Bernice Schacter.
Greenwood Press, 1999
Librarian’s tip: Chap. 9 "Gene Therapy: Magic Bullet or Hype?"
Gene Future: The Promise and Perils of the New Biology
Thomas F. Lee.
Plenum Press, 1993
Librarian’s tip: Chap. 6 "Gene Therapy"
From Alchemy to IPO: The Business of Biotechnology
Cynthia Robbins-Roth.
Perseus, 2000
Librarian’s tip: Chap. 10 "Gene Therapy"
HUMAN GENE THERAPY: Harsh Lessons, High Hopes
Thompson, Larry.
FDA Consumer, Vol. 34, No. 5, September 2000
Better Health through Gene Therapy
Grace, Eric S.
The Futurist, Vol. 32, No. 1, January-February 1998
Genetic Knowledge: Human Values and Responsibility
Jacquelyn Ann K. Kegley.
ICUS, 1998
Librarian’s tip: "Ethical Issues in Human Gene Therapy" begins on p. 176
The Hope, Hype & Reality of Genetic Engineering: Remarkable Stories from Agriculture, Industry, Medicine, and the Environment
John C. Avise.
Oxford University Press, 2004
Librarian’s tip: Discussion of gene therapy begins on p. 149
The Human Genome Project: Cracking the Genetic Code of Life
Thomas F. Lee.
Plenum Press, 1991
Librarian’s tip: "Gene Therapy" begins on p. 289
Genetic Anomaly or Genetic Diversity: Thinking in the Key of Disability on the Human Genome
Iozzio, Mary Jo.
Theological Studies, Vol. 66, No. 4, December 2005
Peer-reviewed publications on Questia are publications containing articles which were subject to evaluation for accuracy and substance by professional peers of the article's author(s).
Genetic Research: Are More Limitations Needed in the Field?
Sosnowski, Kristie.
Journal of Law and Health, Vol. 15, No. 1, Spring 2000
The New Healers: The Promise and Problems of Molecular Medicine in the Twenty-First Century
William R. Clark.
Oxford University Press, 1997
Librarian’s tip: Chap. 9 "Gene Therapy for Cancer"
Technology and Innovation in Japan: Policy and Management for the Twenty-First Century
Martin Hemmert; Christian Oberländer.
Routledge, 1998
Librarian’s tip: Chap. 10 "R&D in Japan's Pharmaceutical Industry: The Biological Revolution, Gene Therapy, and Public Policy"
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