What is Gene Therapy?

Gene therapy is a therapeutic technique involving the insertion of genes into an individual’s cells and tissues, to correct abnormal gene function and help treat disease. This technique allows us to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery. Several approaches to gene therapy are currently in trial, including:

• Substituting a malfunctioning gene with a healthy copy 

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

• Inserting a new gene into the body to help combat disease.

Gene therapy presents a hugely promising treatment option for a number of diseases such as: cancer, cystic fibrosis, heart disease, diabetes and a whole range of inherited diseases. However, there are still risk factors present and clinical trials are underway to make sure they will be safe and effective.

Explore Mayo Clinic studies testing new treatments, interventions and tests as a means to prevent, detect, treat or manage this disease.

Other ongoing gene therapy trials can be found here.