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CMCC 1130

Mouse Model for Drug Development and Research in: (1) Myelination Abnormalities such as ALS (Amyotrophic Lateral Sclerosis), Schizophrenia, Bipolar Disorder; and (2) Drug Addiction

Inventors: Gabriel Corfas

Invention Types: Research Tool, Therapeutics

Research Areas: Neurology/Neuroscience, Psychiatric Disease

Keywords: Animal Model (mouse), Drug Discovery, Drug Screening, Small Molecule/Drug

For More Information Contact:  Caron, Connie

 

Invention Description:

Dr. Gabriel Corfas at Boston Children's and his group are leaders in the field of Neuregulin-ErbB receptor signaling. They were the first ones to link this pathway to oligodendrocyte abnormalities, which are related to defects in the brain white matter and susceptibility to schizophrenia and bipolar disorder. This laboratory develops animal models to study mechanisms underlying neurological disorders, and the effect of treatments on those diseases. |

The group has developed a transgenic mouse in which the erbB signaling pathway is blocked in oligodendrocytes by using the expression of a dominant negative erbB receptor (DN-erbB4), in vivo. The transgenic animals show phenotypes which are counterparts of human neuropsychiatric diseases:

• Changes in oligodendrocyte number and morphology in Central and Peripheral Nervous System

• Reduced myelin thickness

• Slower velocity of conduction in the flow of information to the CNS

• Severe dopaminergic alterations

• Behavioral alterations (abnormal social behavior, locomotor abnormalities, higher anxiety, etc.)

Applications:

Disease Model

Drug Discovery

Drug Screening

Research Tool

Competitive Advantages:

Evolving field linking myelination abnormalities and neuropsychiatric disorders. Dramatic phenotype in the animals, with abnormalities easy to visualize and relatively easy to quantify. Powerful proof of principle model, because of the fundamental relationship of ErbB signaling in the maintenance of oligodendrocytes.

Business Opportunity:

• Non-exclusive licenses with companies developing treatments for amyotrophic lateral sclerosis (ALS)
• Non-exclusive licenses with companies developing treatments for neuropsychiatric disorders
• Non-exclusive licenses with companies developing treatments for drug addiction
• Non-exclusive licenses with companies interested in distributing animal models
• Collaborations and sponsored research

Key Publications: Roy K, Murtie JC, El-Khodor BF, Edgar N, Sardi SP, Hooks BM, Benoit-Marand M, Chen C, Moore H, O'Donnell P, Brunner D, Corfas G. Loss of erbB signaling in oligodendrocytes alters myelin and dopaminergic function, a potential mechanism for the pathogenesis of neuropsychiatric disorders, Proc Natl Acad Sci USA 2007; 104: 8131-8136.

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