Paying close attention to how a canary learns a new song has helped scientists open a new avenue of research against Huntingtons disease a fatal disorder for which there is currently no cure or even a treatment to slow the disease.
In a paper published Sept. 20 in the Journal of Clinical Investigation, scientists at the University of Rochester Medical Center have shown how stem-cell therapy might someday be used to treat the disease. The team used gene therapy to guide the development of endogenous stem cells in the brains of mice affected by a form of Huntingtons. The mice that were treated lived significantly longer, were healthier, and had many more new, viable brain cells than their counterparts that did not receive the treatment.
While its too early to predict whether such a treatment might work in people, it does offer a new approach in the fight against Huntingtons, says neurologist Steven Goldman, M.D., Ph.D., the lead author of the study. The defective gene that causes the disease has been known for more than a decade, but that knowledge hasnt yet translated to better care for patients.
There isnt much out there right now for patients who suffer from this utterly devastating disease, said Goldman, who is at the forefront developing new techniques to try to bring stem-cell therapy to the bedside of patients. While the promise of stem cells is broadly discussed for many diseases, its actually conditions like Huntingtons where a very specific type of brain cell in a particular region of the brain is vulnerable that are most likely to benefit from stem-cell-based therapy.
The lead authors of the latest paper are Abdellatif Benraiss, Ph.D., research assistant professor at the University, and former post-doctoral associate Sung-Rae Cho, Ph.D., now at Yonsei University in South Korea.
The latest results have their roots in research Goldman did more than 20 years ago as a graduate student at Rockefeller Universi
|Contact: Tom Rickey|
University of Rochester Medical Center