Seminar: Stem cells to treat muscular dystrophies
Prof Jennifer Morgan, Emeritus Professor in the Dubowitz Neuromuscular Centre, University College London Great Ormond Street Institute of Child Health
Friday, 08 December 2023, 11am to 12pm
Institute of Developmental and Regenerative Medicine (IDRM) IMS-Tetsuya Nakamura Building Old Road Campus, Roosevelt Drive, Oxford, OX3 7TY
Hosted by Professor Dame Kay Davies
MDUK OXFORD NEUROMUSCULAR CENTRE SEMINAR SERIES
ATTENDANCE
This seminar is intended for the personnel and students at the University of Oxford and the Oxford University Hospitals Foundation Trust.
Hybrid seminar at the IMS-Tetsuya Nakamura Building (IDRM) or on Teams. For the online link, please email chelsea.larabee@idrm.ox.ac.uk.
Students, postdoctoral researchers, and other trainees will be invited to have lunch with Prof Morgan following the talk - register here.
ABSTRACT
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.
SPEAKER
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.
