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

A new study shows that gene therapy can significantly improve hearing in people born with congenital deafness or severe hearing loss.

From Science Daily • Apr. 3, 2026

In theory, the gene therapy works by introducing extra copies of the KL gene into the body’s cells so they make more klotho protein.

From Slate • Mar. 30, 2026

Carrie said: "Grayce's age when she was diagnosed, she couldn't get gene therapy, which would have been a one-off and she probably would have been making her milestones."

From BBC • Mar. 22, 2026

UniQure said earlier this month that the FDA had recommended the company run a new Phase 3 trial for its gene therapy that includes a controlled sham surgery.

From MarketWatch • Mar. 9, 2026

I watch her run back toward the gene therapy room, and then I keep walking.

From "Allegiant" by Veronica Roth