Professor Jacques P. Tremblay has obtained a PhD in Neurosciences from Univ. of California in San Diego (UCSD). He has been working on the development of cell and gene therapies for hereditary diseases (mainly Duchenne muscular dystrophy and Friedreich's ataxia) since 1987. He has published 305 articles in peer-reviewed journals. He is very committed to the clinical application of his research work, as indicated by the conduct, in collaboration with a group of clinicians, of a Phase I clinical trial on 9 Duchenne patients. This trial demonstrated that transplantation onto normal allogeneic myoblasts led to the presence of the normal gene in muscle fibers. He received the award for best researcher in Quebec and the award for best researcher in Canada presented by Muscular Dystrophy Canada for his work on DMD. The Royal College of Physicians and Surgeons of Canada and the Canadian Society for Clinical Investigation presented me with the Henry Friesen Award. He is currently using CRISPR/Prime editing technology to correct point mutations in the APP, DMD, RYR1 and NKX6-2 genes. His research led to the obtention of 5 patents.
My overall research interest is in the area of skeletal muscle in health and disease. The research of my group focuses on various aspects of skeletal muscle biochemistry in health and disease, using exercise and disease models in humans, as well as animal models. In particular, we measure proteins in segments of individual fibres allowing issues with the heterogeneity of skeletal muscle to be overcome. We also examine movement of proteins following micro-dissection of fibres, allowing us to quantitatively assess the redistribution of proteins following various interventions.
Casimir is a Contract Research Organization (CRO) that develops novel outcomes for decentralized and hybrid trials in order to better understand disease progression and treatment benefit. Casimir engages patient communities as partners in research, starting every project from the patient perspective and taking new approaches to understanding and documenting disease progression and treatment benefit.
Prof Watt is Chair of the Department of Anatomy and Physiology at the University of Melbourne and leads the Metabolism and Diabetes laboratory. His team seeks to identify how defects of lipid metabolism and inter-tissue communication cause obesity-related disorders, and to use this information to discover novel targets that can be transitioned to clinical therapeutics.