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Rapamycin Strikes Again

Blocking MTOR Slows Mitochondrial Disease

By Anette Breindl
Science Editor

Wednesday, November 20, 2013
Scientists have shown that inhibiting mTOR more than doubled the life expectancy of mice with Leigh syndrome, a disease that is most often fatal in children by the time they reach school age. That discovery “really runs counter to what has been the prevailing clinical opinion,” Matt Kaeberlein, of the University of Washington, told BioWorld Today. Mitochondria produce the cells’ energy currency, ATP. When mitochondria are not functioning correctly, ATP production is impaired, which impairs cells’ ability to function. The assumption has been that the way to deal with mitochondrial disorders is to “push more calories into the system.”

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