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Grant - Summer 2017 - DMD – Feng Yue, Ph.D.

"Our study will first provide critical insights into the mechanistic basis by which direct inactivation of PTEN in muscle cells benefits the function of dystrophic muscle,” Feng Yue says. “More importantly, we aim to develop a potent pharmacological approach to target PTEN in dystrophic muscle, which could directly lead to the development of novel therapeutic applications for clinical treatment of DMD.”
Feng Yue, research associate scientist at Purdue University in West Lafayette, Ind., was awarded an MDA development grant totaling $175,409 over three years to evaluate the therapeutic potential of a protein called phosphatase and tensin homolog (PTEN) in Duchenne muscular dystrophy (DMD).
In DMD, muscles are more susceptible to injury but they cannot keep up with repair, which eventually leads to muscle loss and weakness.
With colleagues, Yue aims to develop potential therapies that promote regrowth of dystrophic muscle and, in turn, increase muscle strength. The team has developed a strategy that involves inhibiting the action of a natural protein that limits muscle cell growth, called PTEN.
In healthy muscle, the level of PTEN is very low, but it’s found in much higher levels in DMD-affected muscles.
Working with a preclinical mouse model of DMD, the team will first study whether inhibiting PTEN in muscle cells could boost muscle growth and increase muscle strength in DMD mice. The team will then work to develop a safe, high-efficiency pharmacological approach to specifically deliver a well-known PTEN inhibitor to the skeletal muscle of DMD mice, and examine its effect on muscle functional recovery.
If successful, Yue’s work could lead to the development of novel therapeutic strategies for clinical treatment of DMD.
Grantee: DMD – Feng Yue, Ph.D.
Grant type: Development Grant
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