About Gene Therapy

Gene therapy is a therapeutic delivery of nucleic acid polymer into a patient’s cell as a drug to treat disease. Gene therapy could be a way to fix a genetic problem at its source. Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery. Although gene therapy is a promising treatment option for a number of diseases including inherited disorders, some types of cancer, and certain viral infections, the technique remains risky and is still under study to make sure that it will be safe and effective. Gene therapy is currently only being tested for the treatment of diseases that have no other cures. 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 and introducing a new gene into the body to help fight a disease.