London, ON – Six researchers from around the globe have been announced as recipients of over $1 million in funding from London based Defeat Duchenne Canada, The Foundation for Gene and Cell Therapy.
The cutting edge research includes projects in the United States, Toronto and the United Kingdom with continued funding support in Quebec, Toronto and Ottawa.
This represents a total funding investment internationally of more than $9 million in the past 22 years towards discovering a lifesaving cure for Duchenne muscular dystrophy.
In Canada, Defeat Duchenne Canada is excited to partner with Dr. Patrick Gunning and The Gunning Group to fund a study to determine the effectiveness of STAT3 inhibitors in Duchenne muscular dystrophy. This study will be the first of its kind – to date, STAT3 inhibitors have not been researched in Duchenne. Dr. Gunning has partnered with Dr. Michael Rudnicki, Senior Scientist at the Ottawa Health Research Institute. Together, they will aim to develop a molecule to inhibit STAT3 function in Duchenne with a goal of identifying a therapy that can be tested in future human clinical trials.
Additionally, Dr. Ronald Cohn, principal investigator of the study, Paediatrician-in-Chief and Senior Scientist at SickKids utilizes CRISPR technology to target mutations in the DNA referred to as “splice site mutations”. This type of mutation results in alterations to genes that affect the body’s ability to make specific proteins. The study describes multiple applications of this technology as it relates to altering expression of genes or removing certain genetic material for therapeutic purposes.
Defeat Duchenne Canada – background
As the only Canadian charity dedicated solely to funding research related to Duchenne, we are known and respected internationally, having granted more than $9M to research. It is only through the support of donors, families and communities across Canada and around the world that Defeat Duchenne Canada can continue to fund the most promising research into Duchenne muscular dystrophy. And it is only through the advancement of research that the thousands of children who face Duchenne can have hope of a long life and be inspired to live their lives to the fullest today.
DMD, which mainly affects boys, stems from defects in the gene coding for dystrophin, a protein that helps strengthen and protect muscle fibers. Without dystrophin, skeletal and heart muscles degenerate; people with DMD typically end up in a wheelchair, then on a respirator, and die around age 25. The rare disease usually results from missing DNA or other defects in the 79 exons, or stretches of protein-coding DNA, that make up the long dystrophin gene.
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