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.

Additional Resources:

• What Is Gene Therapy? How Does It Work?  U.S. Food & Drug Administration (Español)
• Genes and Gene Therapy  NIH U.S. National Library of Medicine
• Bloomsbury Genetic Therapies  BGT-NPC for Niemann-Pick Disease Type C (NPC)