Website: http://www.ohri.ca/profiles/dilworth.asp

Coming to Canada from

Fred Hutchinson Cancer Research Center, US

Research involves

Studying the processes by which stem cells develop into various types of functional cells within the body.

Research relevance

The research is helping us understand how mature muscle tissue develops from stem cells, thus enabling new approaches for developing treatments for such diseases as muscular dystrophy.

Finding the Genetic Path: From Stem Cells to Muscle Cells

Duchenne muscular dystrophy affects 1 in 3,300 boys, leading to progressive muscle wasting and eventually death from cardiac and respiratory failure. While there is currently no cure for this common genetic disease, stem cell-based gene therapy is widely regarded as having the most promising potential.

Realizing this potential calls for a much deeper understanding of how stem cells develop into mature muscle, Canada Research Chair Dr. Jeffrey Dilworth has launched a research program into the intricate details of this process. Specifically, Dilworth is studying how the muscle specific pattern of gene expression is established during development, so that it can be reproduced exactly in stem cells for therapeutic purposes.

Stem cells have the potential to be directed to form all the different cell types in the body. As a corollary, they also have the potential to express every gene in the genome. Development of mature muscle from stem cells requires both "turning on" (expressing genes that are characteristic of muscle) as well as "turning off" (inhibiting expression of genes that are specific to other cell types). Using an approach that combines protocols using cell culture systems and the test tube, Dilworth is identifying and characterizing the proteins that act as molecular switches to turn genes "on" and "off" during the multiple steps involved in muscle development.

Knowledge gained from Dilworth's work will have application both in the development of new therapeutics for Duchenne muscular dystrophy as well as other genetic diseases.