Dr. Anderson, PhD is an Assistant Professor of Medicine in the Aab Cardiovascular Research Institute and Center for RNA Biology: From Genome to Therapeutics, at the University of Rochester Medical Center in Rochester, NY.
Dr. Ana Topf is a Senior Research Associate at the Translational and Clinical Research Institute at Newcastle University International Centre for Life in Newcastle upon Tyne, UK. Her research focuses on genetics and neuromuscular disorders.
Professor Jordi Díaz-Manera joined Newcastle University in 2020 where he is working as Professor of Neuromuscular Disorders and as Honorary Consultant Clinical Geneticist with the Newcastle Hospitals NHS Foundation Trust.
Rita Barresi is an academic researcher currently working at San Camillo IRCCS, in Venice, Italy. Rita's research is focused on the study of the biomolecular mechanisms underlying neuromuscular diseases and the prevention and recovery of muscle damage. She is involved in the development of innovative prognostic methods and tests to evaluate neuromuscular degeneration.
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.