Members of the Defeat Duchenne Canada (DDC) team spent the day in Toronto, ON, on February 12, 2026. Nicola Worsfold, Executive Director, and Jennifer DiRaimo, Director of Research and Education, were invited to The Hospital for Sick Children, Peter Gilgan Centre, to learn how organizational grant funding is leading towards advancements in Duchenne Muscular Dystrophy (DMD) treatment through the use of a gene editing tool.
DDC began funding Dr. Ronald Cohn’s gene editing research in 2018 and has since then funded over $1 million to The Hospital for Sick Children towards groundbreaking research led by Dr. Cohn and Zhenya Ivakine, PhD. Their research focuses on assessing the effectiveness of using a gene editing tool called CRSIPR/Cas-9 to correct specific DMD pathogenic variants, known as duplications, in mice with a humanized version of the DMD gene.
CRSIPR/Cas-9 corrects the humanized DMD gene variant duplication in mice by ‘cutting it out’ creating a more functioning dystrophin protein. The researchers had to first create a ‘humanized’ mouse with DMD duplication variants to test how will the gene editing tool could work using a ‘humanized’ genetic code. Since most individuals with DMD are treated with steroids to help manage their symptoms, the researchers also ‘pre-treated’ the humanized mice with steroids before using the CRISPR/Cas-9 duplication removal therapy to create a study that more closely resembles what happens in humans. The results from this study will enhance our understanding of how CRSIPR/Cas-9 interacts with drugs commonly used to treat DMD, such as prednisone and vamorolone, highlighting the importance of advancing this therapy into the clinic to improve patient care.
Nicola and Jennifer received a tour of the Ivakine lab and were able to meet with the team of researchers carrying out this important work. Data analysis will be underway soon, and we are looking forward to learning about the results of this study, and next steps.
This initial work has been made possible through funding by DDC which would not be possible without our incredible donors. If you are interesting is supporting our research program, make a donation today.
