Gene therapy is a medical approach that treats or prevents disease by correcting the underlying genetic problem. Gene therapy techniques allow doctors to treat a disorder by altering a person’s genetic makeup instead of using drugs or surgery.
The earliest method of gene therapy, often called gene transfer or gene addition, was developed to:
Later studies led to advances in gene therapy techniques. A newer technique, called genome editing (an example of which is CRISPR-Cas9), uses a different approach to correct genetic differences. Instead of introducing new genetic material into cells, genome editing introduces molecular tools to change the existing DNA in the cell. Genome editing is being studied to:
Gene therapies are being used to treat a small number of diseases, including an eye disorder called Leber congenital amaurosis and a muscle disorder called spinal muscular atrophy. Many more gene therapies are undergoing research to make sure that they will be safe and effective. Genome editing is a promising technique also under study that doctors hope to use soon to treat disorders in people.
MedlinePlus offers a list of links to information about genes and gene therapy.
The Genetic Science Learning Center at the University of Utah provides an interactive introduction to gene therapy and a discussion of several diseases for which gene therapy has been successful.
The Centre for Genetics Education provides an introduction to gene therapy and other therapeutics, including a discussion of ethical and safety considerations.
The National Heart, Lung, and Blood Institute describes the approaches to gene therapy and how they work.
Your Genome from the Wellcome Genome Campus provides an introduction to gene therapy.
KidsHealth from Nemours offers a fact sheet called Gene Therapy and Children.
The information on this site should not be used as a substitute for professional medical care or advice. Contact a health care provider if you have questions about your health.
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