In the News
Gene replacement therapy that works in dogs to be tested in children
"This is a huge deal. This is probably the most important thing we'll ever work on," said Dr. Casey Childers of U.W. Medicine.
For the past eight years, Childers has been focused on finding a cure for a form of muscular dystrophy called Myotubular Myopathy or MTM. It is a rare disease that affects the skeletal muscles.
Now a treatment for MTM that has saved the lives of dogs and is just months away from being tested in children. Dogs involved in the reserach show no signs of the disease after a single infusion of gene-replacement therapy. The disease is so deadly in children, 50-percent of them die before their second birthday.