Editor's Choice Main Category: Huntingtons Disease Also Included In: Stem Cell Research;Neurology / Neuroscience Article Date: 29 Jun 2012 - 14:00 PDT

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At present, there is no cure for the disease and no treatments are available. These findings open up the possibility of testing treatments for the deadly disorder in a petri dish.

The study is the work of a Huntington's Disease iPSC Consortium, including researchers from the Johns Hopkins University School of Medicine in Baltimore, Cedars-Sinai Medical Center in Los Angeles and the University of California, Irvine, and six other groups.

Huntington's disease is an inherited, deadly neurodegenerative disorder. The onset of HD generally occurs during midlife, although it can also strike in childhood - as in the patient who donated the material for the cells generated in this study. The disease causes jerky, twitch-like movements, lack of muscle control, psychiatric disorders and dementia, and ultimately death.

Christopher A. Ross, M.D., Ph.D., a professor of psychiatry and behavioral sciences, neurology, pharmacology and neuroscience at the Johns Hopkins University School of Medicine and one of the lead researchers of the study, explained:

The team are currently testing small molecules for the ability to block HP iPSC degeneration. According to the researchers, these molecules could potentially be developed into new drugs for Huntington's disease.

Furthermore, the teams ability to create "HD in a dish" may also have implications for similar research in other diseases such as Parkinson's and Alzheimer's.

In the study, the team took a skin biopsy from a 7-year-old patient with very early onset of severe HD. In the laboratory of Hongjun Song, Ph.D., a professor at Johns Hopkins' Institute for Cell Engineering, the skin cells were grown in culture and then created into pluripotent stem cells. In addition, a second cell line was created in the same way in Dr. Ross's lab from an individuals without HD.Simultaneously, other HD and control iPS cell lines were generated as part of the NINDS funded HD iPS cell consortium.

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Brain Cells Derived From Skin Cells For Huntington's Research