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

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

Regeneron will offer a new gene therapy for free in the U.S. and invest $27 billion in U.S.

From Barron's • Apr. 23, 2026

"This research highlights not only the potential of gene therapy to change what's possible for children with inherited retinal disease, but also the importance of developing age-appropriate outcome measures."

From BBC • Apr. 22, 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

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

From "Allegiant" by Veronica Roth