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

It’s the second acquisition for BioMarin of 2025, which is seeking to diversify its product portfolio as it recovers from the commercial failure of a hemophilia A gene therapy.

From Barron's

This first-of-its-kind gene therapy, known as BE-CAR7, relies on base-edited immune cells to attack types of T-cell leukemia that historically could not be treated effectively.

From Science Daily

Professor Waseem Qasim, who led the research and is professor of cell and gene therapy at UCL and honorary consultant immunologist at GOSH, said: "We previously showed promising results using precision genome editing for children with aggressive blood cancer and this larger number of patients confirms the impact of this type of treatment. We've shown that universal or 'off the shelf' base-edited CAR T-cells can seek and destroy very resistant cases of CD7+ leukemia."

From Science Daily

He takes a swipe at his predecessor, Peter Marks, for overruling career staff to approve Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy, a debilitating childhood disease.

From The Wall Street Journal

“Drug discovery is just one part,” Visser said in the video, noting that AI can lead to lower medical costs and longer life spans thanks to the “confluence of gene therapy, robotics and diagnostics accelerating.”

From Barron's