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 FDA in recent days has rejected Regenxbio’s gene therapy for the ultra-rare disease Hunter syndrome—which causes deformities and results in death in teenagers—despite the company following to a tee the agency’s guidance to win approval.

From The Wall Street Journal

Elevidys was expected to be transformative for the company, but the gene therapy’s progress has been derailed by a series of safety issues including patient deaths.

From Barron's

As patients and doctors weigh treatment options, the latest data “will surely serve as an important tool, particularly given the one-time nature of a gene therapy,” said Cantor Fitzgerald analyst Kristen Kluska.

From Barron's

While safety is a factor worth monitoring, Sarepta earns some points for being the only gene therapy company to have multi-year data from a “thoughtful number of patients,” with over 1,200 treated to date, Kluska added.

From Barron's

Shares of the rival gene therapy makers fell 0.7% and 1.8%, respectively, on Monday.

From Barron's