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Gene Therapy What is Gene Therapy? • Defective genes make non-functional proteins, creating genetic disorders • Gene therapy corrects defective genes by inserting a functional gene somewhere into the affected chromosome • Gene therapy can also repair bad RNA How Gene Therapy Works • Viruses are genetically altered to carry therapeutic human genes • Virus is a “vector” for the good genes • Virus infects target cell by injecting its DNA or RNA into cell’s genome • Therapeutic genes are incorporated into target cell’s genome, and correct proteins are made How Gene Therapy Works Uses for Gene Therapy • Ex: Severe Combined Immune Deficiency (Bubble Boy) Syndrome - patients don’t make enzyme needed for immune system development - send therapeutic genes to bone marrow cells (make immune system cells) - bone marrow now can make immune cells, and boy no longer lives in bubble David Vetter, Bubble Boy Uses for Gene Therapy • Huntington’s Disease: send in RNA that interferes with and destroys faulty RNA • Hemophilia, Cystic Fibrosis, Thalassemia: substitute faulty mRNA with good mRNA • Instead of fixing adults, we could do gene therapy on sperm, eggs, and embryos (designer babies!) Problems with Gene Therapy • Short term fix (must get continued viral injections) • Immune system can respond to viral vector, killing patient • Only fixes disorders caused by one genewhat about multi-gene disorders? Current Status • FDA has not approved use of gene therapy in U.S. • Based on trial done in France that killed two boys, including David Vetter (bubble boy), in 1999 and 2003. • Is the risk worth the lives that could be saved?