Neurometabolic disease: Treating mitochondrial diseases with mTOR inhibitors - A potential treatment for Leigh syndrome?

摘要

Inhibition of the mechanistic target of rapamycin (mTOR) signalling pathway slows ageing in model organisms. Using a mouse model, researchers from the University of Washington in Seattle now show that mTOR inhibition might also slow the progession of Leigh syndrome, a mitochondrial disease. Every year in the USA, Leigh syndrome affects 1 in 40,000 newborn babies who develop a complex neuropathy and do not live past 6 or 7 years of age.