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

The company plans to refile its application for its gene therapy for a fatal brain disease after the FDA dropped demands to give a placebo to some study subjects.

From The Wall Street Journal • Jun. 23, 2026

Regenxbio went ahead testing Navsunli in 13 subjects, all of whom were given the one-time gene therapy.

From The Wall Street Journal • Jun. 22, 2026

Shares of the Amsterdam-based biopharmaceutical company rose 81% to $48.77 on Wednesday after uniQure said it would seek regulatory approval to bring its gene therapy candidate, AMT-130, to market.

From Barron's • Jun. 17, 2026

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

I leave the gene therapy room just as Nita is walking back to it.

From "Allegiant" by Veronica Roth