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MCL 3344.0: G-Substrate for Treatment and Prevention of Parkinson's Disease

Ole Isacson, M.D. Ph.D., et al.

Background and Description

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 protein G-substrate are neuroprotective of dopaminergic neurons. In vivo rat experiments showed that increasing G-substrate levels in dopaminergic A9 neurons by injection of a viral vector encoding G-substrate resulted in significant neuroprotection of dopaminergic A9 neurons.

Potential Commercial Uses

This invention features methods for the treatment or prevention of PD by increasing the expression or activity of G-substrate (e.g. introducing G-substrate protein or nucleic acid into the A9 region of the brain) or by similarly modulating certain downstream targets of G-substrate in key signaling pathways. 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.

Publication and Patent Status

U.S. and PCT patent applications claiming this invention have been filed. The research underlying the invention has been published as Chung et al. J Neurosci.; 27(31):8314-23 (2007).

Licenses Available

McLean Hospital is offering a worldwide exclusive license to this technology.

For more information, please contact:

David J. Glass, Ph.D.
Senior Associate Director, Technology Transfer
McLean Hospital Research Administration
115 Mill Street
Belmont, MA 02478-9106
(617) 855-3825 - tel
(617) 855-3745 - fax