gene therapy

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.

gene therapy

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

gene therapy

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.

gene therapy

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.)

Word History and Origins

Origin of gene therapy¹

First recorded in 1970–75

Example Sentences

But while Marley, who was diagnosed at five months, cannot walk or talk and needs help to breathe, Meadow is meeting all the milestones for her age after she was tested at birth and received life-changing gene therapy at the earliest opportunity.

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Marley was given Zolgensma, a life-changing gene therapy drug, at 16 months.

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The go-ahead, which came late Monday, will allow Novartis to keep building on what has been a rare commercial success in the troubled field of gene therapy.

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Zolgensma remains virtually the only gene therapy to have proven a true commercial success.

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A three-year-old boy has astounded doctors with his progress after becoming the first person in the world with his devastating disease to receive a ground-breaking gene therapy.

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