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SELECTBIO Conferences Stem Cells in Drug Discovery 2015

Francesco Saverio Tedesco's Biography

Francesco Saverio Tedesco, Principal Research Associate, University College London

I graduated in Medicine and Surgery in 2006 at the University of Rome “La Sapienza” (Italy). During and after medical school I had the opportunity to be a visiting scientist at the Institut Pasteur (Paris, France) in the laboratory of Prof. Shahragim Tajbakhsh, were I studied asymmetric cell division and self-renewal of muscle stem cells. I then continued my studies in the laboratory of Professor Giulio Cossu at San Raffaele Scientific Institute (Milan, Italy), where I obtained my PhD in 2010. After my PhD I coupled post-doctoral research in muscle regeneration with clinical practice and training between Italy (San Raffaele Hospital, Milan) and UK (UCL and UCL Hospitals, London). During this period I also became one of the investigators of a first-in-man clinical trial based upon transplantation of donor muscle stem cells in children with Duchenne muscular dystrophy (DMD; EudraCT n° 2001-000176-33).

I am currently a principal investigator in the UCL Department of Cell and Developmental Biology. My research is focused on the study of muscle regeneration, using murine models of muscular dystrophy and stem cell transplantation as a model system. I am also developing novel therapeutic strategies for muscle disorders by means of human artificial chromosomes, induced pluripotent stem (iPS) cells and biomaterials. I am interested in understanding how skeletal muscle sustains tissue regeneration and how we can improve this process for incurable diseases such as muscular dystrophies.

Our work was the first to show safe and efficacious pre-clinical gene replacement therapy with a human artificial chromosome into an animal model of a genetic disease (specifically, Duchenne muscular dystrophy). We were also amongst the first to report on the therapeutic potential of disease-specific (i.e. limb-girdle muscular dystrophy 2D) iPS cells that have been genetically corrected, transplanted back into an ad hoc generated mouse model and shown to provide therapeutic potential. I am working towards the clinical translation of these strategies into future therapeutic tools for muscle disorders.

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iPS Cells for Muscle Disorders: From Regenerative Medicine to Disease Modelling

Tuesday, 2 June 2015 at 16:45

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I will discuss hurdles for the development of therapies for muscle disorders. I will present our platforms based upon iPS cells, inducible myogenesis and human artificial chromosomes. Extension of these technologies for tissue engineering and drug development will also be discussed.

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