Stem Cells may bring new treatment for Huntington’s Disease

Huntington's disease is a debilitating congenital neurological disorder without effective treatment. But, a special type of brain cell forged from stem cells could help restore the muscle coordination deficits that cause the uncontrollable spasms characteristic of the disease. "This is really something unexpected," says Su-Chun Zhang, a University of Wisconsin-Madison neuroscientist and the senior author of the new study, which showed that locomotion could be restored in mice with a Huntington's-like condition. In the laboratory, Zhang and his colleagues have learned how to make large amounts of GABA neurons from human embryonic stem cells. The goal of this study, Zhang notes, was simply to see if the cells would safely integrate into the mouse brain. To their astonishment, the cells not only integrated but also project to the right target and effectively reestablished the broken communication network, restoring motor function.