Dysregulation of Down syndrome cell adhesion molecule (Dscam) expression has been implicated in many neurological and mental disorders including Down syndrome, Fragile X syndrome, bipolar disorder and intractable epilepsy. Particularly, increased Dscam protein levels have been found to induce overgrowth of axon terminals in developing neurons, which is a major contributor to the pathogenesis of these disorders. There are currently no effective treatments for these neurological disorders, and scientists are only beginning to understand the Dscam regulated pathway.
Novel mediator of Dscam induced aberrant axonal growth
Investigators at the University of Michigan have identified a protein that is a critical mediator of the Dscam induced aberrant axonal growth. In Drosophila melanogaster, mutations in this protein mediator completely suppressed axonal overgrowth due to Dscam overexpression without affecting Dscam expression levels itself, suggesting the protein is essential for Dscam induced axonal overgrowth. Moreover, the investigators indentified an inhibitor of this protein that was able to effectively reduce its activity and Dscam induced axonal overgrowth in Drosophila larvae. This exciting finding could provide a potential therapeutic to treat these currently untreatable neurological and mental disorders.
- Novel target for neurological and mental disorders
- Novel therapeutic for neurological and mental disorders
- Protein inhibitor therapeutic is already FDA approved for treatment of other disorders
- Protein inhibitor therapeutic has been shown to cross the blood-brain barriers, making it effective for treatment of neurological disorders.