Category Archives: Gene Therapy

Please choose from the following list of questions for information about gene therapy, an experimental technique that uses genetic material to treat or prevent disease. On this page: 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 patients 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 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. MedlinePlus from the National Library of Medicine offers a list of links to information about genes and gene therapy. Educational resources related to gene therapy are … Continue reading →

Stanford University researcher Irving Weissman explains how the drug Rituxan, generically called rituximab, improves the cancer-killing effect of a new antibody that renders cancer cells vulnerable to immune attack. He spoke Monday, April 7, at the American Association for Cancer Research meeting in San Diego. The war on cancer is getting some potent reinforcements, including a potentially broad-spectrum new weapon and genetically engineered immune cells with improved cancer-fighting abilities, speakers said at a major cancer research conference held this week in San Diego. The American Association for Cancer Research, attended by an estimated 18,000 participants, is being held at the San Diego Convention Center through Wednesday. While it is covering the gamut of research, cancer immunotherapy is a major focus. The field began more than 100 years ago, and has lately scored impressive advances by using gene therapy to its tool kit. The weapon is an antibody that makes a wide range of cancer cells vulnerable to immune attack. It's close to entering human clinical trials, said Irving L. Weissman, a Stanford University professor leading that project. The antibody neutralizes a chemical signal many cancers exude to decoy the immune system, Weissman said in a Monday morning plenary session. The … Continue reading →

Gene therapy introduces or alters genetic material to compensate for a genetic mistake that causes disease. It is hoped that gene therapy can treat or cure diseases for which no other effective treatments are available. However, many unique technical and ethical considerations have been raised by this new form of treatment, and several levels of regulatory committees have been established to review each gene therapy clinical trial prior to its initiation in human subjects. Ethical considerations include deciding which cells should be used, how gene therapy can be safely tested and evaluated in humans, what components are necessary for informed consent, and which diseases and/or traits are eligible for gene therapy research. Germ Line Versus Somatic Cell Gene Therapy Virtually all cells in the human body contain genes, making them potential targets for gene therapy. However, these cells can be divided into two major categories: germ line cells (which include sperm and eggs) and somatic cells. There are fundamental differences between these cell types, and these differences have profound ethical implications. Gene therapy using germ line cells results in permanent changes that are passed down to subsequent generations. If done early in embryologic development, such as during preimplantation diagnosis and … Continue reading →