There is no cure for the fatal and devastating childhood onset, X-linked disorder, Duchenne Muscular Dystrophy (DMD). Many groups, including my own, are evaluating novel and sometimes sophisticated treatments, either with a view to replace the absent protein, dystrophin, or to mitigate against the deleterious effects of its loss. Mice fail to recapitulate many relevant phenotypic features of the disease, in particular the functional deficits that define the disorder. Larger animal models (including dogs) can help bridge the divide between early evaluations conducted in mice and clinical trials in humans. In this talk I will discuss the founding of the DE50-MD canine model of DMD, maintained at the Royal Veterinary College, studies conducted that make this arguably the best characterised large animal model of this disorder and our use of the model in clinical veterinary trials, including in gene editing and gene therapies. I will end by a discussion of the lengths we go to for optimal welfare and the approach we take to ensure robust and ethical animal use.
SPEAKER
Professor Richard Piercy qualified as a veterinary surgeon from Cambridge University and after a stint in private veterinary practice, he moved to the USA to undertake specialist clinical veterinary training, becoming a Diplomate of the American College of Veterinary Internal Medicine. Whilst in the USA, he also conducted a Master of Science degree on muscle exercise physiology of Alaskan Sled dogs competing in the annual Iditarod race and was a research scholar working on the kinetics of single skinned muscle fibres with Prof. Jack Rall at Ohio State University. Richard moved back to the UK, working as a Wellcome Trust Research Training Fellow for his doctoral training at the Dubowitz Neuromuscular Centre, Imperial College, working on the molecular biology of Emery Dreifuss Muscular Dystrophy, supervised by Prof Francesco Muntoni and Dr Susan Brown. Since 2005, Richard has worked at the Royal Veterinary College where he is now Professor of Comparative Neuromuscular Disease. He runs the largest neuromuscular biopsy service for veterinary species in Europe and the Comparative Neuromuscular Disease Laboratory which researches naturally occurring veterinary neuromuscular diseases and their treatments.
