A groundbreaking and controversial gene therapy experiment is underway at New York Presbyterian Hospital. They're transplanting billions of copies of a gene into the brain of a 55-year-old man with Parkinson's Disease. The procedure is designed to help quiet an area of the brain that is overactive in Parkinson's patients and causes uncontrolled movements and stiffness. Researchers first insert the gene called "Gad" into a weakened, harmless virus that serves as the delivery system. The genes are then injected into the brain through a long thin catheter while the patient is awake. It's hoped that the gene will trigger brain cells to make a chemical called "Gaba" which will help reset the overactive cells.
The FDA has approved a small study of 12 patients to determine if the procedure is safe in people. In studies so far, it has worked but only in monkeys.
We know kids inherit risk factors for things like heart disease, cancer, diabetes but scientists know very little about the triggers that kick start those illnesses. So, now, the government is about to launch the largest, most expensive, long-range study of kids yet, a $2.5 billion project that will follow 100,000 kids for an unprecedented 21 years. Even before the kids are born, researchers will begin by looking at any chemicals or medications taken during pregnancy. Then later, it will follow these children through adulthood, more than two decades. That's just phase one of this effort. Pediatric Cardiologist Edward Clark says that it is an extraordinary partnership between the national institutes of health, the CDC and the Environmental Protection Agency to finally come to an understanding of how environment interacts with genetics and produces health or disease. enrollment for this monumental 21 year study is expected to begin sometime next year.