Therapeutic Potential of a "Membrane Bandage"
The muscle membrane is key to muscle survival. Different types of muscular dystrophy cause the cell membrane to be damaged in different ways. Dysferlin deficiencies show good muscle performance until the late teens or early twenties, when muscle damage becomes critical and results in the onset of muscular dystrophy. A substance called poloxamer 188 has been shown to have protective properties in mouse heart muscle in a different form of muscular dystrophy. This work will explore if and how poloxamer 188 could protect cell and animal models of Miyoshi.
The Jain Foundation also provided a supplemental grant to Dr. Bushby's lab for the purchase of equipment for measuring the mechanical properties of muscles in experimental animals (the In Situ Muscle Test System). This equipment will allow detailed analysis of muscle degeneration and of the benefits of drugs, like poloxamer 188, in animal models of all types of muscular dystrophy.