We study muscle regeneration to develop novel therapies for severe neuromuscular diseases.
We are interested in understanding how stem cells control regeneration of muscles, a complex and very abundant tissue. In particular, we study how muscle regeneration could be improved to develop treatments for severe and incurable childhood diseases like muscular dystrophies.
Our earlier work pioneered the use of human artificial chromosomes and pluripotent stem cells for developing gene and cell therapies for muscle disorders. More recently, we have been investigating the use of small molecules to improve muscle stem cell delivery and the application of novel stem cell-derived artificial muscles for complex disease modelling and tissue engineering.
There are three main things that we will be working on at the Crick:
- Finding out more about the molecular mechanisms of muscle degeneration and regeneration
- Improving our artificial muscle models to help us develop new treatments
- Maximising the therapeutic potential of muscle stem cells for gene and cell therapies
Our ultimate goal is to develop new treatments using these regenerative strategies for children with severe neuromuscular diseases.