Treating Duchenne Muscular Dystrophy Focus of New Research Partnership

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

“Jesse’s Journey is proud to partner with Muscular Dystrophy Canada to fund these two exceptional research studies,” said Perry Esler, Executive Director Jesse’s Journey. “For nearly 25 years, Jesse’s Journey 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 Jesse’s Journey – 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.

– 30 –