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CMCC 1133

Mouse Model for Drug Development and Research in Peripheral Sensory Neuropathies

Inventors: Gabriel Corfas

Invention Types: Research Tool, Therapeutics

Research Areas: Neurology/Neuroscience, Pain

Keywords: Animal Model (mouse), Disease Model, Drug Discovery, Drug Screening

For More Information Contact:  Caron, Connie

 

Invention Description:

Dr. Gabriel Corfas' laboratory is well known for developing animal models to study mechanisms underlying neurological diseases, and the effect of treatments on those diseases. They were leaders in establishing the role of Neuregulin-ErbB receptor signaling in adult peripheral nerves.

Following this discovery, they developed a transgenic mouse model that express the dominant-negative ErbB4 receptor, blocking the ErbB4 pathway in non-myelinating Schwann cells and supporting cells in the inner ear, under the control of the promoter of the Glial Fibrillary Acid Protein (GFAP) gene (named gfa2). The transgenic animals present the following phenotypes:
• Severe peripheral C-Fiber sensory neuropathy with loss of thermal nociception
• Hearing loss due to the primary sensory auditory neuropathy
• Loss of balance due to the vestibular neuropathy

Applications:

Drug discovery, drug screening, research and preclinical assessment of drugs to treat:
• Neuropathies associated with diabetes chemotherapy, HIV, shingles, Fabry's disease, etc.
• Hearing loss and deafness due to sensory auditory neuropathy

• Pain or pain sensitivity Also useful for:
• Gain-of-function studies in cell therapy, to introduce stem cells to replace damaged neurons in specific cases of deafness and loss of balance.

Competitive Advantages:

Dramatic phenotype, with abnormalities easy to visualize and quantify. Powerful proof of principle model, because of the fundamental relationship of ErbB signaling in the maintenance of sensory and Schwann cell functions.

Business Opportunity:

• Non-exclusive licenses with biopharma companies
• Non-exclusive licenses with companies interested in distributing animal models
• Collaborations and sponsored research

Key Publications: Chen S, et al. Disruption of ErbB receptor signaling in adult non-myelinating Schwann cells causes progressive sensory loss. Nat Neurosci. 2003 Nov;6(11):1186-93.

IPStatus: Not applicable