A Philadelphia College of Osteopathic Medicine faculty member who specializes in basic and translational science research for spinal
bulbar and muscular atrophy (SBMA), also known as Kennedy's disease, is propelling
efforts to model and identify therapeutic targets for this neuromuscular disease with
two new grants.
Through awards from the National Institutes of Health (NIH) and the Kennedy’s Disease Association, Heather L. Montie, PhD, an associate professor of Neuroscience, Physiology and Pharmacology, will continue her research efforts to identify potential therapeutic targets for SBMA.
This adult-onset, X-chromosome-linked neuromuscular disease causes progressive difficulty with walking, speaking and swallowing in men. There’s pressing need for such studies because there is currently no cure or effective treatment for SBMA.
The NIH grant will support the creation of a new SBMA zebrafish model that stands to expedite identification of molecular mechanisms of SBMA pathogenesis and discovery or validation of therapeutic interventions.
Since SBMA shares properties with other neuromuscular and neurodegenerative diseases, the ultimate hope is that therapeutic interventions identified for SBMA may also be applicable to or help support therapeutic discoveries for other disorders.
“Creating this new zebrafish model of SBMA is aimed to enable higher throughput studies in our research program and to support more collaborative projects with other SBMA researchers,” Montie said.
The project will also facilitate student research, as the accelerated speed of the modeling is conducive to research projects that would allow PCOM doctor of osteopathic medicine or biomedical sciences students to obtain meaningful results in a few weeks or months, as opposed to longer timeframes that other models can take.
The second grant from the Kennedy’s Disease Association supports Montie’s investigation of the role the CD38 protein has in metabolic dysregulation in SBMA. This funding will support ongoing studies in Montie’s lab to identify potential therapeutic interventions for SBMA.
Ultimately, with new and hopefully better modeling of SBMA, Montie hopes that future research could test for disease-modulating genes or screen libraries of drugs or compounds that one day may help limit the devastating effects of this disease.
This work is supported by the NIH (1R03NS135339) and the Kennedy’s Disease Association.
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