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Yeast model offers clues to possible drug targets for Lou Gehrig's disease, study shows
Date:10/28/2012

STANFORD, Calif. Amyotrophic lateral sclerosis, also called Lou Gehrig's disease, is a devastatingly cruel neurodegenerative disorder that robs sufferers of the ability to move, speak and, finally, breathe. Now researchers at the Stanford University School of Medicine and San Francisco's Gladstone Institutes have used baker's yeast a tiny, one-celled organism to identify a chink in the armor of the currently incurable disease that may eventually lead to new therapies for human patients.

"Even though yeast and humans are separated by a billion years of evolution, we were able to use the power of yeast genetics to identify an unexpected potential drug target for ALS," said Aaron Gitler, PhD, an associate professor of genetics at Stanford. "Many neurodegenerative disorders such as ALS, Parkinson's and Alzheimer's exhibit protein clumping or misfolding within the neurons that is thought to either cause or contribute to the conditions. We are trying to figure out why these proteins aggregate in neurons in the brain and spinal cord, and what happens when they do."

In 2008, Gitler received a New Innovator award from the National Institutes of Health to use yeast as a model for understanding human neurodegenerative diseases and as a way to identify new targets for drug development.

Gitler is the co-senior author of the research, which will be published online Oct. 28 in Nature Genetics. Robert Farese, Jr., MD, a senior investigator at the Gladstone Institutes, is the other co-senior author. Stanford graduate student Maria Armakola shares co-first authorship with Matthew Higgins, PhD, a postdoctoral scholar at Gladstone.

Most cases of ALS have no clear-cut cause. However, it has recently been shown that an RNA-binding protein called TDP-43 accumulates in clumps in the cytoplasm of spinal cord neurons in many people with the condition, and mutations in this protein have been found in some people with the ALS. Researchers li
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Contact: Krista Conger
kristac@stanford.edu
650-725-5371
Stanford University Medical Center
Source:Eurekalert

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