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Potential New Strategy against Huntington’s Disease Found

By R&D Editors | April 13, 2009

Researchers from the MassGeneral Institute for Neurodegenerative Disease (MGH-MIND) have identified a new strategy for removing the abnormal protein that causes Huntington’s disease (HD) from brain cells. This finding may help lead to ways to slow the progression of the devastating neurological disorder. In a study, researchers describe how altering the mutated form of the huntingtin protein appears to accelerate its breakdown and removal through normal cellular processes. 
 
‘One of the major challenges of research into neurodegenerative disorders like Huntington’s, Alzheimer’s and Parkinson’s diseases – all of which involve accumulation of proteins within the brain – has been how to activate degradation machinery that only removes the disease-causing proteins and leaves normal proteins untouched,’ says Dimitri Krainc, MD, PhD, of MGH-MIND, who led the study. ‘We identified a mechanism whereby modification of the disease-causing protein itself facilitates the cell’s method of digesting and recycling the mutant protein.’ 
 
HD is an inherited disorder in which deposits in the brain of the abnormal huntingtin protein lead to degeneration and death of brain cells. The MGH-MIND team found that a standard protein modification process called acetylation appears to flag mutant huntingtin molecules for removal from brain cells without affecting the normal version of the protein. In both cellular and animal models, acetylation of mutant huntingtin also improved neuronal function and prevented neurodegeneration.

Release Date: April 10, 2009
Source: Massachusetts General Hospital

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