What is human gene therapy?
Gene therapy is a treatment using experimental techniques to prevent diseases. In the future, this technique may have the possibility to allow doctors to treat a disorder with no cure by inserting a gene into a patient's cells instead of using drugs or surgery. Scientists at our lab are testing several approaches to gene therapy, including:
- Replacement of a mutated gene that causes the disease
- Inactivation of a mutated gene that is not working properly
- Introduction of a new gene into the body to help fight a disease