The Duchenne Research Fund has awarded a £200,000 grant to the team at the Hospital for Sick Children in Toronto, Canada (‘Sick Kids’), who are working on using CRISPR/Cas9 technology to correct duplication mutations for Duchenne.

Sick Kids’ incoming President and CEO Dr Ronald Cohn has already had significant success as the first researcher to correct duplications in human cells in the lab, using CRISPR technology. Dr Cohn (pictured, far right), Daria Wojtal (centre) and their team have since been working to achieve the same results in mice that have Duchenne duplications, and have so far seen exciting results. The aim is to translate this into boys and young men with Duchenne in the future.

Although CRISPR has been widely used as a research tool, its potential in far-reaching therapeutic applications has largely been unexplored, explains Dr Cohn. “CRISPR is the most important technology that I have encountered in my scientific career thus far. Working with patients and families with genetic disorders, I’m often in a position where I can provide a diagnosis, and perhaps supportive care, but no treatment. CRISPR could change that. It could revolutionise the way we care for patients with currently untreatable genetic conditions.”

Click here to read more about this project.

Click here to read about the team’s previous successes.