gene therapy

American

noun

Medicine/Medical.

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 British

noun

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

"Collins English Dictionary — Complete & Unabridged" 2012 Digital Edition © William Collins Sons & Co. Ltd. 1979, 1986 © HarperCollins Publishers 1998, 2000, 2003, 2005, 2006, 2007, 2009, 2012

gene therapy Scientific

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 Cultural

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.)

Etymology

Origin of gene therapy

First recorded in 1970–75

Example Sentences

Examples are provided to illustrate real-world usage of words in context. Any opinions expressed do not reflect the views of Dictionary.com.

Tilly was part of a gene therapy clinical trial at Sheffield Children's Hospital in 2024 to try to help reduce seizures connected to her condition.

From BBC

The Disc rejection came on the heels of the FDA’s rejection of Regenxbio’s gene therapy for Hunter Syndrome based on similar Prasad reasoning.

From The Wall Street Journal

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

Definitions and idiom definitions from Dictionary.com Unabridged, based on the Random House Unabridged Dictionary, © Random House, Inc. 2023