The Gladstone team will provide DNA samples of HD patients and family members who are unaffected or at risk for HD, which will be sequenced by ISB in partnership with Complete Genomics of Mountain View, CA.
The Gladstone team is collecting DNA samples with help from collaborators including Dr. Vicki Wheelock and Terry Tempkin at the University of California, Davis, Andrea Zanko and Drs. Gail Kang, Michael Geschwind, and Bruce Miller at the University of California, San Francisco, and members of the Northern California Huntington's Disease Society of America. The sequencing project, which is also funded by ISB's Luxembourg program, is the largest complete human genome disease association study conducted to date.
Taube-Koret Center investigators at Gladstone are currently part of a large consortium funded by the National Institutes of Health that uses iPS cells to develop human neurons with HD characteristics. Fibroblasts are obtained from the skin cells of HD patients and converted first into iPS cells and then into neurons that may provide a more accurate platform for testing new therapies than currently available experimental models.
"One of the challenges of Huntington's and many other neurological diseases is that many potential therapies that show promise in animal models turn out to be ineffective in people," Finkbeiner said. "One of the promises of iPS technology is to be able to develop models from HD patients that can give us more detailed information about the disease and better predict how therapies could work in humans."
He pointed out that genomic information from the new collaboration could expand therapeutic opportunities not only for HD but also for other neurological diseases. "The hope is that the work we do here will lay the foundation for applying these techniques to more common but unfortunately more complex neurodegenerative diseases," Finkbein
|Contact: Valerie Tucker|