PerspectiveParkinson’s Disease

A Lysosomal Lair for a Pathogenic Protein Pair

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Science Translational Medicine  13 Jul 2011:
Vol. 3, Issue 91, pp. 91ps28
DOI: 10.1126/scitranslmed.3002808

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Parkinson’s disease (PD) is a progressive neurodegenerative disorder that affects movement. Although many of the causes of PD remain unclear, a consistent finding is the abnormal accumulation of the protein α-synuclein. In a recent issue of Cell, Mazzuli et al. provide a molecular explanation for the unexpected link between PD and Gaucher’s disease, a glycolipid lysosomal storage disorder caused by loss of the enzyme glucocerebrosidase (GBA). They report a reciprocal connection between loss of GBA activity and the accumulation of α-synuclein in lysosomes that establishes a bidirectional positive feedback loop with pathogenic consequences. Understanding how lysosomes are implicated in PD may reveal new therapeutic targets for treating this disease.


  • Citation: T. M. Dawson, V. L. Dawson, A Lysosomal Lair for a Pathogenic Protein Pair. Sci. Transl. Med. 3, 91ps28 (2011).

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