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http://newsblog.mayoclinic.org/2008/11/17/potential-new-therapy-to-stop-progression-of-parkinsons-disease/

Mayo Clinic researchers have developed a method to reduce the production of 
alpha-synuclein in the brain. Alpha-synuclein is a protein that is believed 
to be central to the cause of Parkinson’s disease. All patients with 
Parkinson’s disease have abnormal accumulations of alpha-synuclein protein in 
the brain.
The new method involves the delivery of RNA interference compounds directly to 
selected areas of the brain via injection. The RNA interference compounds 
silence the gene that produces alpha-synuclein, according to the Mayo 
researchers at the Jacksonville, Fla. and Rochester, Minn. campuses.
“While our research has not yet been tested on humans, we expect that these 
findings will lead to an effective treatment for slowing or even halting the 
progression of Parkinson’s disease,” says Demetrius Maraganore, M.D., a Mayo 
Clinic neurologist.
Previous studies conducted by Dr. Maraganore and Matthew Farrer, Ph.D., a Mayo 
Clinic neuroscientist, found that variations in the alpha-synuclein gene 
result in increased protein production and are sufficient to cause 
Parkinson’s disease in some families, or otherwise increase the risk for 
Parkinson’s disease across populations worldwide. Drs. Maraganore and Farrer 
invented a method to treat Parkinson’s disease by reducing alpha-synuclein 
expression. Mayo Clinic patented and licensed their invention to Alnylam 
Pharmaceuticals, Inc. Alnylam is leading the effort to commercialize the Mayo 
invention using Alnylam RNA interference compounds.
“For this study, we developed a lead compound of small interfering RNAs,” says 
Heather Melrose, Ph.D., a Mayo Clinic neuroscientist and a lead author of 
this study. “By infusing this into the brains of mice we were able to 
effectively reduce the production of alpha-synuclein in the brain. The 
therapy produced gene silencing that lasted up to three weeks after 
treatment, and the mice exhibited no ill effects. These are desirable 
characteristics of a drug therapy ultimately intended to treat disease in 
humans.”
The study was published this month in Molecular Neurodegeneration. The full 
news release can be viewed at: 
http://www.mayoclinic.org/news2008-rst/5087.html.

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