A New Target For Machado Joseph Disease Treatment

Machado-Josephdisease (MJD) is a hereditary neurodegenerative disorder that destroys the brainareas involved in muscle control. Although the disease is clearly caused by a mutationin the ATXN3 gene - resulting in an abnormal ataxin-3 protein that forms toxic aggregatesin the brain - the mechanism how MJD develops is unclear. And despite decadesof research no cure or treatment has been found. But now a study in the journal Brain by researchers from Universityof Coimbra in Portugal used a new approach to this old problem, and discovereda way to revert the disease’s neural damage and its symptoms in several animal models of MJD.

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