MDA awarded a research grant totaling $280,487 over a period of two years to Ilona Skerjanc, a professor in the department of biochemistry, microbiology & immunology at the University of Ottawa in Ontario, Canada.
The funds will help support Skerjanc’s investigation into using cell therapies to enhance muscle repair in any of a number of muscle diseases, including congenital muscular dystrophy (CMD); Duchenne (DMD) and Becker (BMD) muscular dystrophies; and Emery-Dreifuss muscular dystrophy (EDMD).
Several cell sources, including satellite cells (a type of immature muscle cell) and mesenchymal stem cells (derived from bone marrow), are currently under study for potential use in therapies to reverse muscle degeneration and strengthen existing muscle. However, Skerjanc noted, difficulties with these approaches include the requirement for invasive procedures, the availability of suitable donors and a limited potential for long-term benefit.
Skerjanc and colleagues recently showed that human embryonic stem (hES) cells can mature into skeletal muscle via progenitor and myoblast (both immature muscle cell types) stages.
In her new work, Skerjanc will isolate skeletal muscle progenitors from hES cells and examine their ability to engraft into skeletal muscle in a mouse model of DMD.
“The overall goal is to provide a method of hES cell differentiation (maturation) and enrichment that will generate human myoblasts and progenitors for long-term engraftment and future therapeutic applications,” Skerjanc said.
Funding for this MDA grant began February 1, 2012.