What is gene therapy?
Gene therapy is an experimental technique that uses genes to treat or prevent diseases that have no cure. This promising 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 Niemann-Pick, the technique is still under study to make sure that it will be safe and effective.