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Treating Duchenne muscular dystrophy focus of new research partnership

April 5, 2019

Toronto, Ontario – Muscular Dystrophy Canada (MDC) and The Foundation for Gene & Cell Therapy (Defeat Duchenne Canada) are joining forces to accelerate ground-breaking research focused on new treatments for Duchenne muscular dystrophy to the sum of $600,000.

Through this partnership, MDC will provide $300,000 with Defeat Duchenne Canada matching the commitment. Two research projects will be funded:

Dr. Anthony Gramolini: Non-viral, immune-modulatory nanoparticles for delivery of CRISPR/Cas9 as a treatment intervention for Duchenne Muscular Dystrophy. Dr. Michael A. Rudnicki: Exosomal Delivery of Wnt7a for Treating Duchenne Muscular Dystrophy.

“For anyone impacted by a neuromuscular disorder, research offers hope that better treatments will be uncovered, and ultimately, a cure. Both of these research projects show very promising initial results and we are thrilled that this investment will help move the research forward,” said Barbara Stead-Coyle, CEO, Muscular Dystrophy Canada. “We are thrilled to be working with Defeat Duchenne Canada to fund this promising research and can’t wait to see what positive impacts it has on our clients and families.”

Both projects focus on improving a critical component, the delivery vehicle, used in gene therapies like gene transfer and gene editing.

“Defeat Duchenne Canada is proud to partner with Muscular Dystrophy Canada to fund these two exceptional research studies,” said Perry Esler, Executive Director Defeat Duchenne Canada. “For nearly 25 years, Defeat Duchenne Canada has funded the most promising Duchenne research worldwide and we’ve seen first-hand the difference that is made in the life of a person facing Duchenne when they are able to benefit from scientific discovery. Jesse Davidson – the inspiration behind Defeat Duchenne Canada – once said, “When researchers work together, miracles can happen.” We believe the same can be true when funding organizations work together, and it’s in the spirit of collaboration we welcome the opportunity to join forces with MDC.”

There are already several clinical trials exploring gene transfer (a type of gene therapy) underway for Limb-Girdle and Duchenne Muscular Dystrophies. These make use of AAV (adeno-associated virus) as the transport vehicle but limitations exist in using AAVs including small packaging capacity, difficulty in producing large amounts, and potential to cause an immune response especially if they need to be administered more than once.

To circumvent some of the limitations of AAV, both research projects will be testing alternative non-viral vehicles.

Dr. Gramolini and his team will test BIPHIP, a nanoparticle which will be used to transport CRISPR gene editing tools into cells. with the goal of editing a genetic change that causing this disorder. Dr. Rudnicki and his team will test exosomes as vehicles to deliver the Wnt7a gene, which has been shown to help muscle cells regrow.

If successful, the results could have broader applicability to other types of muscular dystrophies.

About Muscular Dystrophy Canada

Muscular Dystrophy Canada’s mission is to enhance the lives of those impacted with neuromuscular disorders by continually working to provide ongoing support and resources while relentlessly searching for a cure through well-funded research. To learn more about Muscular Dystrophy Canada, please visit or call our toll-free number at 1-800-567-2873.

About Defeat Duchenne Canada

Defeat Duchenne Canada is a partnership of science and families dedicated to the discovery of treatment and cure of Duchenne muscular dystrophy – because parents should not out live their children. To learn more about Defeat Duchenne Canada visit or call 519-645-8855.

Media Contact Information

Heather Rice
Muscular Dystrophy Canada
[email protected]