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Gladstone, Stanford scientists block toxic protein that plays key role in Lou Gehrig's disease
Date:10/28/2012

SAN FRANCISCO, CAOctober 28, 2012 Scientists at the Gladstone Institutes and the Stanford University School of Medicine have discovered how modifying a gene halts the toxic buildup of a protein found in nerve cells. These findings point to a potential new tactic for treating a variety of neurodegenerative conditions, including amyotrophic lateral sclerosis (ALS, or Lou Gehrig's disease)a fatal disease for which there is no cure.

The Gladstone and Stanford scientists began their experiments independently before realizing that combining their efforts could strengthen their results. Their discoverywhich involved the work of both neuroscientists and geneticistsunderscores the importance of collaborative and cross-disciplinary research when dealing with complex neurodegenerative diseases such as ALS.

ALS usually strikes between the ages of 40 and 75, ravaging the body's motor neuronsnerve cells that control muscle movement. This causes muscle weakness, difficulty swallowing and breathing, paralysis and, ultimately, deathoften just three to five years after diagnosis. At any given time, as many as 30,000 Americans are living with ALSwhich afflicts physicist Stephen Hawking and which killed baseball legend Lou Gehrig.

In a paper published today online in Nature Genetics, researchers in the laboratories of Stanford Associate Professor Aaron D. Gitler, PhD, and Gladstone Senior Investigators Robert V. Farese, Jr., MD and Steve Finkbeiner, MD, PhD, describe how shutting off a gene called Dbr1 in yeast cells and in neurons obtained from rats can protect both cell types from the toxic effects of TDP-43a protein that plays a key role in ALS.

"Mutations in the gene that produces TDP-43 can cause this protein to build up in cells," said Dr. Farese, who is also a professor at the University of California, San Francisco, with which Gladstone is affiliated. "Over time, TDP-43 accumulation inside motor neurons can reach toxic levels
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Contact: Anne Holden
anne.holden@gladstone.ucsf.edu
415-734-2534
Gladstone Institutes
Source:Eurekalert  

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