gene therapy

American  

noun

Medicine/Medical.

1. the application of genetic engineering to the transplantation of genes into human cells in order to cure a disease caused by a genetic defect, as a missing enzyme.

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gene therapy British  

noun

1. the replacement or alteration of defective genes in order to prevent the occurrence of such inherited diseases as haemophilia. Effected by genetic engineering techniques, it is still at the experimental stage

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gene therapy Scientific  

1. The treatment of a disorder or disease, especially one caused by the inheritance of a defective gene, by replacing defective genes with healthy ones through genetic engineering.

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gene therapy Cultural  

1. A promising technology that involves replacing a defective gene in the body with a healthy one. This can be done by removing cells from the body, using genetic engineering techniques to change defective sequences in the DNA, and then reinserting the cells. This technique has been carried out successfully, for example, on bone marrow cells, in which defective cells were successfully replaced with healthy, genetically engineered cells. Scientists hope to find an agent, such as a therapeutic virus, that will be able to correct defective DNA in situ. (See cloning vector.)

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Etymology

Origin of gene therapy

First recorded in 1970–75

Example Sentences

Prof Simon Jones, a leading expert in inherited metabolic diseases, said gene therapy had been approved in the UK in 2022.

From BBC • Jun. 2, 2026

Ditto a gene therapy by UniQure for the brutal neurodegenerative Huntington’s Disease, which slowed progression by 75% in a clinical trial.

From The Wall Street Journal • May 3, 2026

The company also said it would offer a new gene therapy for free in the U.S., a genetic hearing loss treatment called Otarmeni.

From Barron's • Apr. 23, 2026

Funding came from several Chinese research programs as well as Otovia Therapeutics Inc., the company that developed the gene therapy and employs many of the researchers involved.

From Science Daily • Apr. 3, 2026

We follow her past the gene therapy room where I saw a map of my genetic code, farther into the heart of the compound than I have been yet.

From "Allegiant" by Veronica Roth