Rare genetic disorder provides unique insight into Parkinson's disease
Massachusetts General Hospital investigators appear to have found the mechanism behind a previously reported link between the rare genetic condition Gaucher disease and the common neurodegenerative disorder Parkinson's disease. In a report to appear in the July 8 issue of Cell and receiving early online release, they describe how disruption of the molecular pathway that causes Gaucher disease leads to the toxic neuronal deposits of the protein alpha-synuclein (α-syn) found in Parkinson's and related disorders. In addition, rising α-syn levels further inhibit the Gaucher's-associated pathway, leading to even more α-syn deposition, a finding that indicates therapies targeting this pathway may be a new option for patients with Parkinson's disease.