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New model of ALS is based on human cells from autopsied tissue
Date:8/11/2011

ugs or small molecules, such as the RNA fragments tested in this study, might be used to reduce SOD1 function in astrocytes and suppress their toxicity. Drs. Kaspar and Burghes are developing a viral system that would carry these RNA fragments through the bloodstream and into the spinal cord, and deliver them to astrocytes. This work is being done in collaboration with Don Cleveland, Ph.D., at the University of California, San Diego, as part of the Recovery Act grant. Several labs are pursuing cell replacement therapies for ALS, and the current work adds to evidence that replacing astrocytes may be just as important as, and perhaps easier than, replacing motor neurons lost to the disease.

"It's been a long road, but the hard work starts now," Dr. Kaspar said. "We still need to confront fundamental questions about what is happening to astrocytes and how they are killing motor neurons. And the ultimate goal is to identify therapies that will translate into helping humans."


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Contact: Daniel Stimson
stimsond@ninds.nih.gov
301-496-5751
NIH/National Institute of Neurological Disorders and Stroke
Source:Eurekalert

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