gene therapy

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Discover how gene therapy can treat diseases caused by genetic mutations such as cystic fibrosis

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Science & Tech

gene therapy

medicine

Also known as: gene transfer therapy, genetic modification

Written by John Harris, Catherine Stanton, Anthony J.F. GriffithsSee All

Fact-checked by The Editors of Encyclopaedia Britannica

Last Updated: Mar 22, 2024 • Article History

Category: Science & Tech

Also called:: gene transfer therapy

Key People:: Fred H. Gage

Related Topics:: therapeutics; human genetic disease; gene doping; genetic disease

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Recent News

Mar. 21, 2024, 5:43 AM ET (National Institutes of Health (NIH) (.gov))

Experimental gene therapy for giant axonal neuropathy shows promise in NIH clinical trial

Mar. 20, 2024, 6:53 PM ET (News-Medical)

Investigational gene therapy shows promise for rare childhood neurodegenerative disease

gene therapy, introduction of a normal gene into an individual’s genome in order to repair a mutation that causes a genetic disease. When a normal gene is inserted into the nucleus of a mutant cell, the gene most likely will integrate into a chromosomal site different from the defective allele; although that may repair the mutation, a new mutation may result if the normal gene integrates into another functional gene. If the normal gene replaces the mutant allele, there is a chance that the transformed cells will proliferate and produce enough normal gene product for the entire body to be ...(100 of 712 words)

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