I put gene therapy up there with the technology that makes the GPS in my car work. It reminds me of the wonderful quote from Arthur C. Clarke, “Any sufficiently advanced technology is indistinguishable from magic.”
Research is advancing gene therapy as a possible treatment or eventual cure for genetic diseases that bedevil modern science. Gene therapy was conceived over 20 years ago, and until recently, remained largely in the research lab. But gene therapy products are now beginning to be approved by the U.S. Food and Drug Administration for clinical care. Physician-scientists are intrigued with exploring its possibilities for transforming medical practice.
Gene therapy seeks to target faulty genes that are driving disease and either correct or replace them. Imagine your entire genome as an electric master board that controls physical characteristics and bodily functions. A genomic variant would be the burned out fuse causing disease. Gene therapy would target the defective fuse and either replace it or add a new fuse to get the body functioning correctly.
As an example of the potential, David Deyle, M.D., of the Mayo Clinic Department of Clinical Genomics and Center for Individualized Medicine Clinomics Program, is using gene therapy in his research into possible treatments for osteogenesis imperfecta, also known as brittle bone disease. People with this devastating rare genetic disorder suffer with bones that break easily and often. Caused by a defect in the protein known as collagen, brittle bone disease has no cure. Continue reading