Muscles grown in the lab by researchers at the University of Toronto used to study Duchenne muscular dystrophy and develop treatments

Inside a Petri dish in a University of Toronto lab is a muscle – made from scratch using human stem cells – with Duchenne muscular dystrophy (DMD).

To study the biological properties of DMD, a degenerative muscle disease that primarily affects men, researchers at the University of Toronto obtained cell lines from people living with the disease and used them to create miniature muscles in a dish. . Today, they are helping other researchers and industry partners to develop and test new treatments that can help boys and young men with DMD.

The research team is led by Bryan stewart, professor of biology at U of T Mississauga, and Penny gilbert, Associate Professor at the Institute of Biomedical Engineering and the Donnelly Center for Cellular & Biomolecular Research at the University of Toronto. Stewart specializes in the physiology of neurons and muscles. Gilbert, a cell biologist, specializes in restoring skeletal muscles (the muscles attached to bones) using stem cells. They decided to collaborate after meeting at a research leadership workshop hosted by a university professor Molly shoichet about six years ago.

“We learned that we both study skeletal muscles,” says Gilbert. “Bryan’s lab used fruit flies to understand the muscle-nerve connection, which allows the brain to tell, for example, our arm to move.

“My lab was creating human tissue to create models of the muscle-nerve connection. Together, we realized that our unique tools and methods could allow us to look at DMD in a different way than, literally, any band in the world.

DMD is caused by a genetic mutation that prevents the body from making dystrophin, the protein that makes muscles work. It is a rare disease – occurring in one in 3,500 to 5,000 male children worldwide – but it is devastating. From around the age of five, DMD gradually damages and weakens muscles, including the heart. Most children with DMD will need to use a wheelchair. And most will die before they turn 30.

Gilbert says that the biomedical innovation of muscle building “means that for the first time it’s actually possible to study DMD and the nerve-muscle connection outside the body.

“This gives us the opportunity to revisit observations that were made decades ago that seemed to suggest that the muscle-nerve connection in DMD may be impaired,” she says. “Now let’s see if this can be observed in our model. And if we see it, could we try to use it as a starting point to find molecules that could improve the muscle-nerve connection? “

Stewart points out that while the team doesn’t directly create drugs or therapies, their work will be an important foundational system that other researchers can use to develop pharmaceutical treatments or test gene therapy experiments that will soon go into clinical trials. .

Michael Rudnicki, a leading Canadian stem cell expert, agrees.

“The preclinical DMD assays developed by Gilbert and Stewart are an essential facet of the translational pipeline, enabling the testing of current and future therapies in the context of human cells,” said Rudnicki, Principal Scientist and Director of Regenerative Medicine. and the Sprott Center for Stem Cell Research at the Ottawa Hospital Research Institute.

“There is a wealth of excellent work being done on DMD around the world,” says Stewart. “Penney and I knew when we first met at this workshop that there could be a lot of power in merging our two groups.

“I think we are at a point where we can help launch a new wave of testing and discovery that will begin to benefit individuals and families living with DMD.”

A century after the discovery of insulin, the University of Toronto and its hospital and industry partners have built a culture of discovery, innovation and collaboration that has transformed healthcare and continues to have an impact. workout around the world. This article is part of a series featuring researchers working on medical and health innovations for the future.

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