Gene Therapy

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Gene Therapy, experimental medical treatment that manipulates a gene or genes within cells in order to produce proteins that change the function of those cells. Gene therapy originated in efforts to treat and cure some of the more than 9,000 known genetic disorders, most of which lack an effective therapy.

The original goal of gene therapy was to substitute a healthy gene for a defective one, or to repair a faulty gene, thereby eliminating symptoms of disease. But researchers have moved beyond inherited genetic disorders to treat other kinds of diseases. Cancer begins in genes and may be caused by an inherited defect or a mutation (permanent alteration to a gene) that causes a cell to malfunction. AIDS is caused by a virus that disrupts the genetic material of immune cells. Other new gene therapy projects are targeted at conditions such as heart disease, diabetes mellitus, arthritis, and Alzheimer's disease, all of which involve genetic susceptibility to illness. Gene therapists hope to reduce or eliminate this susceptibility. Eventually, gene therapy might help older people to regain strength in withered muscles and density in thinned bones, and to increase pumping power in their aging hearts. Some researchers predict that in the distant future the technology could be used to eliminate genetic defects from families or even to produce “designer babies” with more muscle strength, higher intelligence, sweeter dispositions, or whatever traits parents desire.

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