As part of the $3.7 million grant from the National Institutes of Health, the Cedars-Sinai Regenerative Medicine Institute, directed by Clive Svendsen, Ph.D., will supply scientists at five leading laboratories, including Cedars-Sinai, with all the adult stem cells used for a consortium study to develop potential therapies to treat Huntington’s disease.. The consortium comprises a partnership of five leading Huntington's disease laboratories at Cedars-Sinai, the Gladstone Institutes, Johns Hopkins, Massachusetts General Hospital and the University of California at Irvine.
Huntington’s disease, also known as “Huntington's chorea" and "Woody Guthrie's disease," is an incurable neurodegenerative genetic disorder that affects muscle coordination and some cognitive functions, such as memory. It can also affect personality, causing increased confusion and anger. More than 100,000 Americans and 1 million worldwide have Huntington’s or are at risk of inheriting the disease from a parent. Any child of an affected parent has a 50 percent risk of inheriting the disease.
The study will compare stem cells from patients with Huntington’s with stem cells from healthy patients in an effort to understand why brain cells die in Huntington’s patients, causing uncontrollable body movements and psychological changes. The project will use induced pluripotent stem cell technology. The production of such cells from adult human skin biopsies is at the functional core of the new Cedars Regenerative Medicine Institute facility.
Adapted from the Cedars Sinai announcement.
Comments