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

See Examples For:

gene therapy

UniQure says it still sees a path to regulatory approval for the drug candidate, AMT-130, even as the experimental gene therapy for Huntington’s disease falls hort in a recent study.

From Barron's ● Sep. 29, 2026

Shares of Ultragenyx Pharmaceutical rose 13% to $14.50 after the Food and Drug Administration granted commercial approval to the biotech firm’s gene therapy for pediatric patients with a rare neurodevelopmental disease.

From The Wall Street Journal ● Sep. 17, 2026

Existing approaches include gene therapy, which is suitable for only a small fraction of patients with particular mutations, and electronic retinal prostheses, which can be invasive, costly, and require significant training.

From Science Daily ● Aug. 28, 2026

Her dad Charlie felt earlier detection could have given her a chance of walking, as he had seen "a big difference" since she had a one-off gene therapy infusion in Bristol.

From BBC ● Aug. 9, 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