Methods for the Diagnosis and Treatment of Parkinson's Disease

McLean Hospital
posted on 05/18/2011

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Parkinson’s Disease (PD) is a chronic, progressive motor system disorder affecting as many as one million Americans, that can arise from environmental and/or genetic factors. Regardless of specific etiology, it has been found that dopaminergic neurons in the A9 region of the brain (substantia nigra pars compacta) are considerably more vulnerable than dopaminergic neurons in the immediately adjacent A10 region (ventral tegmental area). It is likely that different gene products expressed in the A10 neurons provide a neuroprotective effect. Based on extensive gene expression studies comparing A9 and A10 neurons, the inventors have discovered that elevated levels of the transcription factor Otx2 are neuroprotective of dopaminergic neurons. Otx2 is a homeodomain transcription factor, whose expression is restricted to the forebrain and midbrain in the nervous system during development and which plays an important role in proliferation and differentiation of midbrain dopaminergic progenitors during mitosis.Otx2 overexpression in A9 neurons of rodents caused increased expression of proteins known to be overexpressed in A10 neurons, and resulted in significant neuroprotection of the A9 neurons.
Potential Commercial Uses: This invention features methods for the treatment or prevention of PD by increasing the expression or activity of Otx2 (e.g. introducing Otx2 protein or nucleic acid into the A9 region of the brain). By identifying a novel target for PD, this invention will lead to safer, more specific, and more powerful treatments for PD, which would be of great importance given the prevalence of this disease in our aging populations. Current therapy for PD, such as the use of the dopamine precursor L-DOPA to replace brain dopamine, has had only limited effectiveness.
Patent Status: A U.S. patent application claiming this invention has been filed. The research underlying the invention has been published as Chung et al. Brain 133(7):2022-31 (2010). (Epub 2010 Jun 23)
Licenses Available: McLean Hospital is offering a worldwide exclusive license to this technology.