Stem cells to treat muscular dystrophies

Hybrid seminar - Lunch with students/postdocs to follow. Email for further info.

Stem cell therapy was first suggested as a promising strategy for treatment of muscular dystrophies such as Duchenne Muscular Dystrophy (DMD) in the 1980s. The aim was to restore the missing protein, dystrophin, in regenerated muscle fibres derived from donor cells. In addition to muscle fibre formation, reconstitution of the functional muscle stem cell pool by donor cells is vital for long-term treatment. Since then, researchers have tested the capacity of different types of stem cell to contribute to skeletal muscle regeneration. We have gained important insights into the control of quiescence, activation and differentiation of muscle stem cells and the impact of a pathological muscle environment on the function of these cells. But before stem cells could be considered for clinical application, there remain several hurdles to be overcome: producing sufficient cells to treat skeletal muscles bodywide, effective systemic delivery of stem cells, producing enough dystrophin in treated muscles to be of functional benefit and prevention of immune rejection of the cells or muscle fibres of donor origin.

Jennifer Morgan is an emeritus professor in the Dubowitz Neuromuscular Centre, University College London Great Ormond Street Institute of Child Health. The main areas of her research are the identification of stem cells that contribute to skeletal muscle regeneration, and the genetic and functional manipulation of these cell populations to enhance muscle repair in skeletal muscle regenerative medicine and in the treatment of muscular dystrophies. She has also been involved in pre-clinical work on the use of antisense oligonucleotides to restore dystrophin expression.