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Brendan Lee, M.D., Ph.D.

Dr. Brendan Lee Appointed Director

The Program Advisory Committee unanimously voted to appoint Baylor pediatric geneticist Brendan Lee, M.D., Ph.D., director of the Bone Disease Program of Texas.

Lee is a professor in Baylor’s Department of Molecular and Human Genetics and is Chief of the Skeletal Dysplasia Clinic at Texas Children’s Hospital. Lee belongs to an elite group of scientists with the title of Howard Hughes Medical Institute (HHMI) Investigator. He was one of 12 physician scientists chosen in 2002 as part of a new HHMI initiative to fund translational research.

He is interested in the consequences of gene mutations on craniofacial and limb development. To understand these consequences, he combines studies on tissue and organ development with clinical research in patients who have skeletal malformations.

Born in Hong Kong, Lee immigrated to the United States at an early age and spent his childhood in Brooklyn and Queens. He worked hard in school, where he especially excelled in math and science. “I was lucky because the things that were ‘acceptable’ in immigrant culture were the things I loved,” Lee said.

At sixteen, he entered a combined BS/MD program at the City University of New York, Brooklyn College/State University of New York Health Science Center at Brooklyn. After completing his BS in chemistry in three years, Lee pursued both an M.D. and a Ph.D. in genetics focusing on collagen defects. As a postdoctoral student at Mt. Sinai, Lee helped to clone a gene for Marfan Syndrome. Marfan Syndrome is a genetic disorder characterized by disproportionately long limbs, long thin fingers and tall stature, often associated with Abraham Lincoln. While he found the genetics lab fascinating, Lee’s strongest motivation was to help patients - many of them children – with genetic skeletal defects like osteogenesis imperfecta (OI), or brittle bone disease. 

OI is a devastating childhood bone disease characterized by a low bone mass at birth. In its most severe form, children die young because of fractures of their rib cage, making it impossible for them to breathe normally. In less severe cases, these youngsters develop fractures of multiple bones during childhood and adolescence, which causes considerable deformities and long-term problems. The majority of children with this disorder have a defect in the type-1 collagen gene that leads to weakness of the skeleton. 

A major portion of Lee’s laboratory work focuses on the role of collagen in skeletal deformities.  Most recently, Lee and colleagues identified a new gene, a mutation of CRTAP, which causes a recessive form of OI in mice and in humans. The devastating effects of OI in children led Lee to establish the Skeletal Dysplasia Clinic at Texas Children’s Hospital and to pioneer the use of bisphosphonates to treat the secondary osteoporosis in these patients.

Lee exemplifies translational research at its best, combining the strengths of laboratory research and patient care. Lee says, “In our skeletal dysplasia rounds we review the patients, look at x-rays, discern patterns, and come up with new hypotheses. We test them in the laboratory and then, hopefully, come back to the patients with a clearer understanding of the nature of the diseases and better options for care.”

The Operating Agreement between Baylor and MD Anderson stipulates rotation of the leadership every five years. Past director, Robert F. Gagel, M.D. will serve as co-director of the program.


© 2014 The University of Texas MD Anderson Cancer Center