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A treatment that modifies the genome to treat or prevent a particular genetic condition or disease.

Use in clinical context

The principle behind gene therapy is that deliberate and specific changes are made to a patient’s DNA to prevent, cure or alleviate a genetic condition. This can be achieved by adding a functional copy of a gene, disabling a gene that makes a faulty product or changing gene activation.

Gene therapy can have long-lasting effects because it permanently changes the cell’s DNA, with these changes being inherited by any daughter cells that result if the cell divides. However, these changes will not be passed on to any children that the patient may have in the future as they are targeted to specific cells that do not include the person’s eggs or sperm.

There are an increasing number of gene therapies available for clinical use and in clinical trials.

Last updated on 1st March, 2023