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Dr. Brendan Lee

Dr. Brendan Lee, professor in the Department of Molecular and Human Genetics at Baylor College of Medicine and Howard Hughes Medical Institute investigator, has been elected to membership in the Institute of Medicine.

Lee is co-director of the Rolanette and Berdon Lawrence Bone Disease Program of Texas, a collaboration of BCM, The University of Texas MD Anderson Cancer Foundation and the newly established Center for Skeletal Medicine and Biology at BCM. 

He holds the Robert and Janice McNair Endowed Chair in Molecular and Human Genetics and is the founder and director of the Skeletal Dysplasia Clinic at Texas Children’s Hospital.

He is one of 70 new members and 10 foreign associates named to the institute today. BCM now has 13 IOM members.

Well-deserved honor

“Dr. Brendan Lee represents the best of translational research,” said Dr. Paul E. Klotman, president and CEO of BCM. “Along with pursuing groundbreaking research that leads to improved patient care, he trains new scientists and physicians and improves the quality of education at the school. His work and his character are typical of those elected to the prestigious Institute of Medicine. It is a well-deserved honor.”

“This is a great honor,” said Lee.  “Ultimately, it reflects the superb environments at Baylor College of Medicine and Texas Children’s Hospital that have allowed our team to translate cutting edge genetics and genomics into clinical practice.”

Research interests

Lee is interested in understanding how gene mutations affect skeletal development. He combines studies in the laboratory that focus on mammalian tissue and organ development with clinical research involving patients with skeletal problems.

He is also interested in disorders of metabolism or energy regulation. For example, he is studying people with urea cycle disorders who cannot remove nitrogen from their blood. By focusing on the relationship between these disorders and nitric oxygen, he hopes to find better treatments, perhaps using the molecule itself or employing gene therapy.

Lee was an M.D./Ph.D. student at State University of New York, Downstate Medical Center in the late 1980s, when he cloned two genes for connective tissue diseases – Marfan syndrome and osteogenesis imperfecta. That sparked a fire in him to complete his M.D. degree along with his Ph.D. and pursue genetics both in the laboratory and the clinic. He completed his pediatric residency and genetic fellowship at Baylor College of Medicine, where he found his academic home.

In 2006, he and his colleagues pinpointed the gene CTRAP as responsible for a previously unidentified form of osteogenesis imperfecta, the so-called “brittle bone disease.”

In 2012, he was able to apply new insight into a child genetic disorder – argininosuccinic aciduria – to a treatment. The disorder resulted in the inability to make a key amino acid, arginine. Treating the child with arginine supplements left him with two symptoms – high blood pressure and neurodevelopmental delay. Lee and his team found that the patient could not use the arginine he took to make nitric oxide.

Lee thought he had an answer. He admitted the child to the hospital and weaned him off his blood pressure medicine. Then he started him on a form of nitrate – the kind of drug people take to relieve the chest pain of angina. The treatment addressed the problem of the lack of nitric oxide directly.

“Having an immediate impact on the life of a child is the most rewarding feeling,” said Lee. This treatment is a graphic example of the translational research Lee hoped to pursue while still in graduate school.

Popular mentor

Lee is known as a mentor and founded and directs the Medical Student Research Track at Baylor that gives medical students who do not want to pursue a Ph.D. the chance to do research in the laboratory. He is also currently a member of the Faculty Operating Committee of the Medical Students Training program (M.D./Ph.D.).

He is a member of the graduate programs in the Departments of Genetics, Cell and Molecular Biology, Developmental Biology and Translational Biology and Molecular Medicine at Baylor College of Medicine. Lee serves on multiple national advisory boards and currently chairs National Institute of Dental and Craniofacial Research Board of Scientific Counselors (BSC).

He is the recipient of many awards, including the 2005 American Philosophical Society’s Judson Darland Prize for Achievement in Patient-Oriented Clinical Research, the 2007 Michael E. DeBakey Excellence in Research Award, the 2009 Edith and Peter O'Donnell Award of The Academy of Medicine, Engineering and Science of Texas and the 2009 E. Mead Johnson Award from the Society for Pediatric Research, Baylor College of Medicine Alumni Association Medical School Young Alumnus Award (2012), and election to the Association of American Physicians (2010), American Society for Clinical Investigation (2008), Alpha Omega Alpha (1989) and Phi Beta Kappa (1986).

A well-recognized scientist, he is the author of more than 170 articles in peer-reviewed journals.

IOM members at Baylor

Dr. Lee joins the following group of distinguished scientists:

Dr. Bobby R. Alford

Dr. Arthur L. Beaudet

Dr. Dennis M. Bier

Dr. William R. Brinkley

Dr. William T. Butler

Dr. C. Thomas Caskey

Dr. Mary K. Estes

Dr. Richard A. Gibbs

Dr. Peter J. Hotez

Dr. James R. Lupski

Dr. Bert W. O’Malley

Dr. Huda Y. Zoghbi