Francesco Saverio Tedesco, Head of the Stem Cells and Neuromuscular Regeneration Laboratory at the Crick, has been awarded the scientific coordination of a €9.5m grant from Horizon Europe and UKRI to lead a ground-breaking international project to find new treatments for muscular dystrophies.
Muscular dystrophies involve muscle weakness and wasting, causing impaired movement and potentially shortening lifespan. Although research has helped identify genetic mutations associated with different types of disease, there still aren’t effective genetic therapies for most muscular dystrophies.
Led by Saverio and his team at UCL and the Crick, the project, called MAGIC, brings together 15 partners across nine countries, from research hospitals and institutions to businesses and charities. Saverio will also work closely with Mario Amendola from Inserm and Genethon in France and group leaders Andrea Serio and Sam Rodrigues at the Crick.
The group’s primary goal is to create human models of skeletal muscle disease, to allow the development of new genetic therapies. These models will be cell-based, with the hope of developing vectors (deactivated viruses) which can be used to deliver gene therapies, such as a healthy copy of a faulty gene or gene editing tools.
Animal models which have been used to study muscular dystrophy have often failed to predict potentially toxic side effects of new gene therapies. The MAGIC project will aim to reduce the use of animal models by creating simplified ‘muscle in a dish’ models which can predict toxicity and how well the therapy will work before it’s tested in people.
The idea will also be to generate more specific therapies for different types of muscular dystrophies. For example, one type involves muscle fibres becoming weak because supporting cells around them are not working properly. Working out how to target these support cells could help re-strengthen the muscle.
Involving biotechnology companies will facilitate translation of key project outputs towards potential treatments as soon as possible. The researchers will also collaborate with people living with muscular dystrophies, through organisations like Muscular Dystrophy UK, ensuring that the voices of people with lived experiences shape the research.
Saverio said, “As a paediatrician, I want to be able to offer hope to the children that I treat and their families. This project represents a significant step forward in the search for effective treatments for muscular dystrophies. Through cutting-edge bioengineering, innovative genetic therapies, and advanced translation, we aim to make a difference in the lives of people affected by these devastating diseases."
Paul Nurse, the Crick’s director, said, “This ambitious project highlights the value of collaborative working across borders and across different scientific areas. I hope that association with Horizon Europe will allow more large-scale international projects like this to flourish.”