The technology provides a novel gene encoding an MNR2 protein that generates motor neurons in vivo. Several neurodegenerative diseases such as ALS result in loss of motor neurons and consequent immobility and death. There is currently no available cure. One possible way to treat neurodegenerative diseases is to regenerate lost motor neurons. The technology provides several ways to accomplish this task, most directly comprising the full MNR2 protein and nucleic acid sequence -- potentially useful in genetic tests and medical diagnostics - as well as methods of producing the protein. The technology includes pharmaceutical compositions used as therapeutics and could also be used in drug discovery applications to identify regions of the protein critical for generation of motor neurons.
While other genes that lead to generation of motor neurons have been identified, they do so indirectly. In contrast, MNR2 is sufficient to drive motor neuron generation -- a rare example in which activation of a single gene may be enough to treat neurodegenerative disease. As a homeobox gene, MNR2 directly binds DNA and regulates the expression of other genes which characterize the division of progenitor cells into differentiated motor neurons. Additionally, nascent motor neurons extend their axons which indicates that the new motor neurons will successfully integrate into existing circuits.
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Patents Issued (US 6,387,656; US 7,074,771, US 7,192,916)
Available for licensing and sponsored research support
Tech Ventures Reference: IR 861