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What is gene therapy?

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including:

  • Replacing a mutated gene that causes disease with a healthy copy of the gene.

  • Inactivating, or “knocking out,” a mutated gene that is functioning improperly.

  • Introducing a new gene into the body to help fight a disease.

Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective. Gene therapy is currently only being tested for the treatment of diseases that have no other cures.

For general information about gene therapy:

MedlinePlus from the National Library of Medicine offers a list of links to information about genes and gene therapyThis link leads to a site outside Genetics Home Reference..

Educational resources related to gene therapyThis link leads to a site outside Genetics Home Reference. are available from GeneEd.

The Genetic Science Learning Center at the University of Utah provides an interactive introduction to gene therapyThis link leads to a site outside Genetics Home Reference. and a discussion of several diseases for which gene therapy has been successfulThis link leads to a site outside Genetics Home Reference..

The Centre for Genetics Education provides an introduction to gene therapyThis link leads to a site outside Genetics Home Reference., including a discussion of ethical and safety considerations.

KidsHealth from Nemours offers a fact sheet called Gene Therapy and ChildrenThis link leads to a site outside Genetics Home Reference..

Additional information about gene therapyThis link leads to a site outside Genetics Home Reference. is available from the National Genetics and Genomics Education Centre of the National Health Service (UK)


Next: How does gene therapy work?

 
Published: April 21, 2014