Online Seminar: Engineering human stem cells for muscle disease modelling and therapy development

This seminar is intended for the personnel and students at the University of Oxford and the Oxford University Hospitals Foundation Trust.

Online Seminar – please register for this Zoom event

Skeletal muscle is the most abundant human tissue. It has a complex structure and some regenerative capacity, supported by a pool of muscle stem cells. Numerous, severe genetic diseases impair skeletal muscle function and regenerative capacity, with the vast majority of them still remaining incurable. Our laboratory studies skeletal muscle regeneration, focusing on the development of novel therapies for incurable neuromuscular disorders of childhood. Our work pioneered the use of human artificial chromosomes and induced pluripotent stem (iPS) cells for gene and cell therapies of muscular dystrophies. Recently, we developed the first 3D artificial skeletal muscle entirely derived from patient-specific induced pluripotent stem (iPS) cells, and we showed that it can model severe forms of muscular dystrophy with high fidelity and resolution. Current projects focus on generating advanced human in vitro models of neuromuscular diseases for therapy development, as well as on using small molecules to improve human muscle stem cell delivery to target tissues. During my talk I will recap and review significant achievements of our previous work, and present new unpublished results on advanced human neuromuscular disease modelling and stem cell engineering for muscle gene and cell therapy.

Prof. Tedesco is a clinician-scientist with expertise in neuromuscular diseases and muscle regeneration. He graduated in Medicine and Surgery with honours at the Sapienza University of Rome (Italy). Before his doctorate he was a visiting scientist at the Institut Pasteur (Paris, France) studying muscle stem cell biology. He obtained his PhD investigating novel gene and cell therapies for muscular dystrophy at the San Raffaele Scientific Institute of Milan (Italy). Dr Tedesco established his research group at UCL in 2014 and in 2015 he received the Young Investigator Award by the European Society of Gene and Cell Therapy. He was then awarded an NIHR Academic Clinical Fellowship, followed by a Clinical Lectureship, and a prestigious €1.5 million European Research Council (ERC) Starting Grant. More recently, he received the 2020 Simon Newell Investigator of the Year award by the Royal College of Paediatrics and Child Health, the 2021 MacKeith Prize by the British Paediatric Neurology Association and was promoted Professor at UCL.

The Tedesco laboratory studies skeletal muscle regeneration, focusing on the development of novel therapies for incurable neuromuscular disorders of childhood. They work pioneered the use of human artificial chromosomes and induced pluripotent stem (iPS) cells for gene and cell therapies of muscle diseases. Recently they developed the first human 3D artificial skeletal muscle entirely derived from iPS cells of children with neuromuscular diseases. Current projects investigate the use of small molecules to improve muscle cell delivery and iPS cell-derived myogenesis for complex neuromuscular disease and therapy modelling. The overall goal of the Tedesco laboratory is the translation of the aforementioned regenerative strategies into novel therapies to improve outcomes for children with neuromuscular disorders.

For more information about Prof. Tedesco's, visit his laboratory website