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School of Medicine
Se-Jin Lee, M.D., Ph.D.
The Michael and Ann Hankin and Partners of Brown Advisory Professor in Scientific Innovation
Professor of Molecular Biology and Genetics
Research Interests: Regulation of mammalian development and adult tissue homeostasis by growth and differentiation factors
Dr. Se-Jin Lee is a professor of molecular biology and genetics at the Johns Hopkins University School of Medicine. He is the Michael and Ann Hankin and Partners of Brown Advisory Professor in Scientific Innovation. Dr. Lee is credited with discovering myostatin, a protein that inhibits muscle growth.
His research focuses on regulation of mammalian development and adult tissue homeostasis by growth and differentiation factors. The primary interest of Dr. Lee’s laboratory is to understand the role of signaling molecules in regulating embryonic development and adult tissue homeostasis.
Dr. Lee received his undergraduate degree in biochemistry from Harvard College. He earned his M.D. and Ph.D. in molecular biology and genetics from the Johns Hopkins University School of Medicine and joined the Johns Hopkins faculty in 1991.
He is a member of Medical Advisory Committee of the Muscular Dystrophy Association. He became a member of the National Academy of Sciences in 2012 and was elected as a fellow of the American Association for the Advancement of Science in 2010. Dr. Lee has more than 50 U.S. patents issued.
- The Michael and Ann Hankin and Partners of Brown Advisory Professor in Scientific Innovation
- Professor of Molecular Biology and Genetics
Departments / Divisions
- M.D., Johns Hopkins University School of Medicine (Maryland) (1989)
- Ph.D., Johns Hopkins University School of Medicine (Maryland) (1989)
Johns Hopkins University School of Medicine, Baltimore, MD, 1981, Medical Scientist Training Program
Research & Publications
Dr. Lee’s primary interest is to understand the role of signaling molecules in regulating embryonic development and adult tissue homeostasis. He has focused on the superfamily of secreted proteins that are structurally related to transforming growth factor-Β (TGF-Β). Members of this growth factor family have been shown to play important roles in regulating the development and function of many different tissues, and as a result, many of these factors have shown enormous therapeutic potential for a wide range of clinical applications. Using molecular genetic approaches, he and his lab have identified a large number of novel mammalian TGF-Β family members that we have designated growth/differentiation factors (GDFs). They have been using a variety of experimental approaches, including genetic manipulation of mice, to attempt to understand the precise biological functions of these molecules. We are particularly interested in understanding the roles of these molecules in regulating tissue growth.
Much of his work has focused on a molecule that he and his team have designated myostatin. They have shown that myostatin is expressed specifically in developing and adult skeletal muscle and that mice engineered to lack myostatin exhibit dramatic increases in skeletal muscle mass throughout the body. Based on these and other studies, they believe that myostatin normally acts to block skeletal muscle growth.
Dr. Lee and his team are currently attempting to elucidate the mechanism of action of myostatin as well as the mechanisms by which the activity of myostatin is regulated. Their long term goal is to attempt to exploit the biological properties of myostatin to develop novel therapeutic strategies for treating patients with muscle degenerative and wasting conditions, such as muscular dystrophy, sarcopenia, and cachexia resulting from diseases like cancer, AIDS and sepsis.
Lab Website: Se-Jin Lee Lab
Se-Jin Lee, Lori A. Reed, Monique V. Davies, Stefan Girgenrath, Mary E.P. Goad, Kathy N. Tomkinson, Jill F. Wright, Christopher Barker, Gregory Ehrmantraut, James Holmstrom, Betty Trowell, Barry Gertz, Man-Shiow Jiang, Suzanne M. Sebald, Martin Matzuk, En Li, Li-fang Liang, Edwin Quattlebaum, Ronald L. Stotish, and Neil M. Wolfman (2005) "Regulation of muscle growth by multiple ligands signaling through activin type II receptors." Proc. Natl. Acad. Sci., USA 102:18117-18122.
Markus Schuelke, Kathryn R. Wagner, Leslie Stolz, Christoph Hubner, Thomas Riebel, Wolfgang Komen, Thomas Braun, James F. Tobin, and Se-Jin Lee (2004) "Gross muscle hypertrophy in a child associated with a myostatin (GDF-8) mutation." New Engl. J. Med. 350:2682-2688.
Teresa A. Zimmers, Monique V. Davies, Leonidas G. Koniaris, Paul Haynes, Aurora F. Esquela, Kathy N. Tomkinson, Alexandra C. McPherron, Neil M. Wolfman, and Se-Jin Lee (2002) "Induction of cachexia in mice by systemically administered myostatin." Science 296:1486-1488.
Se-Jin Lee and Alexandra C. McPherron (2001) "Regulation of myostatin activity and muscle growth." Proc. Natl. Acad. Sci., USA 98:9306-9311.
Alexandra C. McPherron, Ann M. Lawler, and Se-Jin Lee (1997) "Regulation of skeletal muscle mass in mice by a new TGF-ß superfamily member." Nature 387:83-90.
Academic Affiliations & Courses
Graduate Program Affiliation
Biochemistry, Cellular and Molecular Biology (BCMB) Graduate Program
Cellular and Molecular Medicine (CMM)
Human Genetics and Molecular Biology
Activities & Honors
- Fellow, American Association for the Advancement of Science (AAAS), 2010
- Summa Cum Laude, Harvard College, 1981
- Phi Beta Kappa, Harvard College, 1980
- The Michael and Ann Hankin and Partners of Brown Advisory Professorship in Scientific Innovation, 2013
- Ho-Am Prize in Medicine, 2013
- Rolf Luft Award, Karolinska Institutet, 2013
- National Academy of Sciences (Medical Physiology and Metabolism), 2012
- Medical Advisory Committee, Muscular Dystrophy Association, 2010