Shunji Tomatsu is a Professor, Principal Research Scientist, and Director of Skeletal Dysplasia Center Pediatric Orthopedic Surgery, Nemours Children's Health. He is also a Professor at the Department of Pediatrics, Thomas Jefferson University, Philadelphia, PA, and the Department of Pediatrics, St. Louis University, St. Louis, MO. He obtained an MD at the Gifu University School of Medicine, Gifu, Japan and a Ph.D. at the Gifu University School of Medicine, Department of Pediatrics, Gifu, Japan. For over thirty-five years, Tomatsu's group has focused on bench-to-bed diagnosis and treatment for a rare hereditary disease Mucopolysaccharidosis (MPS), especially the type IV MPS (Morquio A syndrome). They identified the disease-related genes, generated animal models, established diagnostic approaches, and developed therapeutic strategies including AAV and lentivirus gene therapies. Finally awarded the FNIH AMP® BGTC grant * (May 2023), to conduct a first-of-its-kind gene therapy clinical trial for Morquio A. They are also engaged in other cutting-edge methods including small molecular therapy. His research interests include mucopolysaccharidosis; skeletal dysplasia; inactive project newborn screening, and targeting therapy.