Robert and Janice McNair Endowed Chair
Professor and Chair, Department of Molecular and Human Genetics
Director, Center for Skeletal Medicine and Biology
Baylor College of Medicine
Managing Co-Director, Rolanette and Berdon Lawrence Bone Disease Program of Texas
Dr. Brendan Lee is Professor and Chair of the Department of Molecular and Human Genetics at Baylor College of Medicine (BCM). Dr. Lee co-directs the joint MD Anderson Cancer Center, University of Texas Health, and BCM Lawrence Family Bone Disease Program of Texas, and the BCM Center for Skeletal Medicine and Biology. He is also the Founder and Director of the Skeletal Dysplasia Clinic at Texas Children’s Hospital.
Dr. Lee has pioneered the translation of gene discoveries into therapies for rare skeletal and metabolic diseases. He identified the first mutations in human dwarfism and Marfan syndrome, and then in the transcriptional factors that regulate these matrix proteins during skeletogenesis. He showed how dysregulation of post‐translational modification of collagens causes osteogenesis imperfecta (OI) leading to the discovery of over 25 new OI genes. Focusing on how matrix regulates bone, he showed that increased TGF-β signaling is a common driver of OI and he is clinically testing this approach. Dr. Lee identified WNT1 as the key WNT regulating human bone mass. He identified Notch dysregulation as a driver of osteosarcoma and breast cancer metastasis. He then developed gene therapy for the osteoarthritis in chondrodysplasias that led to the first in vivo high-capacity adenoviral gene therapy trial in knee osteoarthritis, which recently received FDA RMAT designation.
He leads the NIH BCM Undiagnosed Diseases Network Clinical Site, NIH BCM RE-JOIN Consortium site, and NIH Brittle Bone Disorders Consortium. He holds multiple patents in drug discovery and gene therapy and these licensed technologies are now in industry-sponsored clinical trials (for osteoarthritis, Osteogenesis Imperfecta, and Maple Syrup Urine Disease).
Dr. Lee has been elected to the National Academy of Medicine, American Academy of Arts and Sciences, American Association for the Advancement of Science, the Texas Academy of Medicine, Engineering, Science, and Technology (TAMEST), the Association of American Physicians, the American Society for Clinical Investigation, and the Society of Pediatric Research.
