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August 8, 2017Scientist Awarded AHA Grant to Develop Therapies for Rare Form of Childhood Muscular Dystrophy
Scientist Bijan K. Dey could soon offer hope to young patients with a rare form of muscular dystrophy.
Dey, a faculty researcher at The RNA Institute at the University at Albany, has received the American Heart Association’s (AHA) prestigious Scientist Development Grant to develop a basic understanding and develop therapies for a deadly genetic childhood-muscle degenerative disease known as Duchenne Muscular Dystrophy (DMD).
DMD is a hereditary condition characterized by progressive muscle weaknesses and muscle degeneration. Among nine types of muscular dystrophy, DMD is the most severe and life-threatening. It is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. As the disease progresses, it weakens the heart and respiratory muscles causing serious medical issues.
Symptoms typically begin in early childhood, usually between the ages of 3 and 5. Children with DMD are commonly restricted to wheelchairs within the first decade of their lives with an average life expectancy of 26. It primarily affects young boys.
“DMD currently has no cure. My team is exploring a novel approach for therapy that has not been previously investigated,” Dey said. “Our goal is to gain a better understanding of the pathophysiology of DMD and DMD and associated heart dysfunctions in molecular detail. We are thankful for AHA’s support.”
Dey’s research is focused on a class of small non-coding RNA molecules called microRNAs. During his postdoctoral training at the University of Virginia School of Medicine, he demonstrated that small non-coding RNA molecules regulate the stem cell population crucial to maintaining normal skeletal and cardiac muscle function – or normal human health.
His lab is investigating the underlying molecular mechanisms of how the microRNAs maintain normal muscle function and what goes wrong with these RNAs when children are diagnosed with DMD and associated heart dysfunctions.
Dey is optimistic that through the support of the AHA and The RNA Institute his team can develop a basic understanding of the disease, follow its progression, and provide an avenue for therapy-based treatment.
“Dey’s innovative research program speaks to the vision of The RNA Institute to create translational therapies requiring both basic and applied research on non-coding RNA’s role in muscle development and related human diseases,” said Paul Agris, outgoing director of The RNA Institute and professor of biology at UAlbany. “Dr. Dey has the knowledge and experience to bring a whole new approach to muscular dystrophy research and take it to a new height.”
Marlene Belfort, a distinguished professor at UAlbany and interim director of The RNA Institute, added that Dey has demonstrated his knowledge and expertise by earning highly competitive funding on the national stage. “At this funding climate, it is extremely challenging for a young investigator to be awarded an extramural grant early in their career,” she said.
Dey’s project is funded by the AHA through June 2020 for $231,000. It will not only support his research but also allow for mentor-based training of students and future scientists through The RNA Institute.
