Brendan Lee

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

Chair

(713) 798-5443

Email

blee@bcm.edu

Positions

Chair: Molecular and Human Genetics
��Ƶ of Medicine

Robert and Janice McNair Endowed Chair and Professor: Molecular and Human Genetics
��Ƶ of Medicine
Houston, TX, US

Member: Development, Disease Models & Therapeutics Graduate Program
��Ƶ of Medicine

Member: Genetics and Genomics Graduate Program
��Ƶ of Medicine

Director: Center for Skeletal Medicine and Biology
��Ƶ of Medicine

Director: Skeletal Dysplasia Clinic
Texas Children's Hospital

Associate Director: Medical Research Pathway
��Ƶ of Medicine

Co-Director: Rolanette and Berdon Lawrence Bone Disease Program of Texas
��Ƶ of Medicine

Member: Dan L Duncan Comprehensive Cancer Center
��Ƶ of Medicine
Houston, Texas, United States

Professor: Molecular and Human Genetics
��Ƶ of Medicine

Addresses

Skeletal Dysplasia Center (Clinic): Texas Children's Clinical Care Center
6701 Fannin St., 16th Floor
Houston, TX, 77030
United States
Phone: (832) 822-4298

Education

BS from City University Of New York, Brooklyn College: 06/1986 - Brooklyn, NY, United States

PhD from State University Of New York Downstate Medical Center: 06/1990 - Brooklyn, NY, United States

MD from State University Of New York Downstate Medical Center: 06/1993 - Brooklyn, NY, United States

Residency at ��Ƶ Of Medicine: 06/1995 - Houston, TX, United States; Pediatrics

Clinical Fellowship at ��Ƶ Of Medicine: 06/1997 - Houston, TX, United States; Clinical Genetics

Clinical Fellowship at ��Ƶ of Medicine: 06/1998 - Houston, Texas, United States; Clinical Biochemical Genetics

Post-Doctoral Fellowship at Mount Sinai School of Medicine: 01/1991 - New York, New York, United States

Certifications

Clinical Biochemical Genetics: American Board of Medical Genetics

Clinical Genetics: American Board of Medical Genetics

General Pediatrics: American Board of Pediatrics

Honors & Awards

William F. Neuman Award for Outstanding Scientific, Training, Research, and Administrative Contributions: 2022; American Society for Bone and Mineral Research

Curt Stern Award for Outstanding Scientific Achievement: 2016; American Society for Human Genetics

Fellow AAAS: 2014; American Association for the Advancement of Science

National Academy of Medicine: 2013

Investigator: Howard Hughes Medical Institute (10/2002 - 10/2014)

Association of American Physicians: 2010

E. Mead Johnson Award for “Outstanding Scientific Achievement in Pediatrics”: 2009

American Society for Clinical Investigation: 2008

Edith and Peter O'Donnell Award in Medicine: 2009; Texas Academy of Medicine, Science and Engineering

Michael E. DeBakey Excellence in Research Award: 2007

American Philosophical Society’s Judson Darland Prize for Achievement in Patient-Oriented Clinical Research: 2005

Society for Pediatric Research Young Investigator Award: 2000

Professional Interests

Developmental, translational and clinical studies of skeletal dysplasias and inborn errors of metabolism

Professional Statement

As a pediatrician and geneticist, the overall mission of my research program is to translate the study of structural birth defects and inborn errors of metabolism into a basic understanding of development, disease and novel therapeutic approaches. In the area of metabolism, we have applied genetic approaches to the study of biochemical genetic disorders (specifically urea cycle disorders) as models of complex diseases (those involving nitric oxide dysregulation). This has led us to study the consequences of metabolic derangements broadly in the endocrine, cardiovascular, skeletal, renal and neurological systems. In the area of structural birth defects, we have studied paracrine and endocrine signaling pathways that regulate skeletal development including morphogens (TGF, Wnt and Notch), and extracellular matrix proteins and their modifications (e.g., collagen prolyl-hydroxylation and lysyl-hydroxylation) that contribute to human skeletal dysplasias including brittle bone diseases and connective tissue diseases like Ehlers-Danlos syndrome. These developmental pathways have led us to ask how their dysregulation contributes to common diseases such as osteoporosis, osteoarthritis and bone cancer.

Our mechanistic discoveries are translated via clinical research that is performed as part of the Skeletal Dysplasia Clinic and the Metabolic Disorders Clinic at Texas Children’s Hospital, respectively, and as part of the NIH Rare Diseases Clinical Research Network (RDCRN) Brittle Bone Disorders Consortium. My clinical research program began with stable isotopic measurements in urea cycle disorder patients to better assess new treatments. I have led investigator-initiated studies testing combined phenylbutyrate/arginine treatment and nitric oxide supplementation in patients with argininosuccinic aciduria and phenylbutyrate in maple syrup urine disease and industry-sponsored Phase II and III studies of the now FDA-approved ammonia scavenger glyceryl-triphenylbutyrate in urea cycle patients. In the area of skeletal dysplasias, I have studied the utility of zoledronic acid, teriparatide and anti-TGF treatments in pediatric and adult osteogenesis imperfecta. Our preclinical gene therapy studies have led to a clinical trial of high-capacity adenoviral gene therapy in osteoarthritis. As an extension of these studies, I lead the RE-JOIN consortium of the NIH HEAL initiative mapping the neuronal pain mediators into the osteoarthritis joint. As part of my genomic medicine studies, I have focused on advancing the diagnosis of genetic diseases via gene discovery, multi-omic approaches to phenotyping and mechanistic studies in the NIH Undiagnosed Diseases Network. Finally, I am committed to developing a scientific workforce as part of the All of Us Evenings with Genetics Scholars Program.

Websites

Department of Molecular and Human Genetics

Center for Skeletal Medicine and Biology & Rolanette and Berdon Lawrence Bone Disease Program of Texas

Clinics

Research Laboratory

Selected Publications

Ketkar S, Dai H, Burrage LC, Murdock D, Dawson B, Acosta-Herrera M, Kerick M, Martin J, Wilhelm K, Asmussen JK, Lichtarge O, Center RG, Assassi S, Mayes MD, Lee BH. " " Ann Rheum Dis. 2025 Aug ; 84 : 1363-1374.
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Chen YT, Jiang MM, Leynes C, Adeyeye M, Majano CF, Ibrahim B, Polak U, Hung G, Jin Z, Lanza DG, Liao L, Dawson B, Chen-Everson Y, Ruiz OE, Gibbons RJ, Heaney JD, Bae Y, Lee BH. " " J Clin Invest. 2025 Jan ; 135 : e163587.
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Marom R, Song IW, Busse EC, Washington ME, Berrier AS, (...), Lee BH. " " J Clin Invest. 2024 Jun ; 134 : e170369.
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Song, I. W., Nagamani, S. C., Nguyen, D., Grafe, I., et al., Lee, B.. " " J Clin Invest.. 2022 Apr ; 132 (7) : e152571.
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Marom R, Burrage LC, Venditti R, Clément A, Blanco-Sánchez B, (…), Undiagnosed Diseases Network, Westerfield M, De Matteis MA, Lee B. " " Am J Hum Genet.. 2021 ; 108 : 1710-1724.
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Lim J, Lietman C, Grol MW, Castellon A, Dawson B, (…), Lee BH. " " Proc Natl Acad Sci U S A. 2021 ; 118 : e2100690118.
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Jin Z, Kho J, Dawson B, Jiang MM, Chen Y, Ali S, Burrage LC, Grover M, Palmer DJ, Turner DL, Ng P, Nagamani SC, Lee B. " " J Clin Invest. 2021 Mar 1; 131 (5) : e138935.
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Murdock DR, Dai H, Burrage LC, Rosenfeld JA, Ketkar S, Müller MF, Yépez VA, Gagneur J, Liu P, Chen S, Jain M, Zapata G, Bacino CA, Chao HT, Moretti P, Craigen WJ, Hanchard NA; Undiagnosed Diseases Network, Lee B. " " J Clin Invest. 2021 Jan 4; 131 (1) : e141500.
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Burrage LC, Reynolds JJ, et al, Undiagnosed Diseases Network, Bertola D, Yamamoto GL, Baratela WAR, Butler MG, Ali A, Adeli M, Cohn DH, Krakow D, Jackson AP, Lees M, Offiah AC, Carlston CM, Carey JC, Stewart GS, Bacino CA, Campeau PM, Lee B.. " " Am J Hum Genet.. 2019 ; 104 (3) : 422-438.
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Kho J, Tian X, Wong WT, Bertin T, Jiang MM, Chen S, Jin Z, Shchelochkov OA, Burrage LC, Reddy AK, Jiang H, Abo-Zahrah R, Ma S, Zhang P, Bissig KD, Kim JJ, Devaraj S, Rodney GG, Erez A, Bryan NS, Nagamani SCS, Lee BH.. " " Am J Hum Genet. 2018 ; 103 (2) : 276-287.
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Nixon AJ, Grol MW, Lang HM, Ruan MZC, Stone A, Begum L, Chen Y, Dawson B, Gannon F, Plutizki S, Lee BHL, Guse K. " " Arthritis Rheumatol.. 2018 ; 70 (11) : 1757-1768.
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Joeng KS, Lee YC, Lim J, Chen Y, Jiang MM, Munivez E, Ambrose C, Lee BH. " " J Clin Invest.. 2017 May ; 127 (7) : 2678-2688.
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Zeng HC, Bae Y, Dawson BC, Chen Y, Bertin T, Munivez E, Campeau PM, Tao J, Chen R, Lee BH. " " Nat Commun. 2017 Apr 11; 8 : 15000.
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Egunsola AT, Bae Y, Jiang MM, Liu DS, Chen-Evenson Y, Bertin T, Chen S, Lu JT, Nevarez L, Magal N, Raas-Rothschild A, Swindell EC, Cohn DH, Gibbs RA, Campeau PM, Shohat M, Lee BH. " " J Clin Invest. 2017 Mar 6; 127 (4) : 1475-1484.
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Tao J, Jiang MM, Jiang L, Salvo JS, Zeng HC, Dawson B, Bertin TK, Rao PH, R. Chen R, Donehower LA, Gannon F, Lee B (2014). " " Cancer Cell. 2014 ; 26 (3)
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Grafe I, Yang T, Alexander S, Homan EP, Lietman C, Jiang MM, Bertin T, Munivez E, Chen Y, Dawson B, Ishikawa Y, Weis MA, Sampath TK, Ambrose C, Eyre D, Bächinger HP, Lee B. " " Nat Med.. 2014 Jun ; 20 (6) : 670-5.
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Orwoll ES, Shapiro J, Veith S, Wang Y, Lapidus J, Vanek C, Reeder JL, Keaveny TM, Lee DC, Mullins MA, Nagamani SC, Lee B. " " J Clin Invest.. 2014 Feb 3; 124 (2) : 491-8.
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Laine CM, Joeng KS, Campeau PM, Kiviranta R, Tarkkonen K, Grover M, Lu JT, Pekkinen M, Wessman M, Heino TJ, Nieminen-Pihala V, Aronen M, Laine T, Kröger H, Cole WG, Lehesjoki AE, Nevarez L, Krakow D, Curry CJ, Cohn DH, Gibbs RA, Lee BH, Mäkitie O. " " N. Engl. J. Med.. 2013 May 9; 368 (19) : 1809-16.
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Ruan MZ, Erez A, Guse K, Dawson B, Bertin T, Chen Y, Jiang MM, Yustein J, Gannon F, Lee BH. " " Sci Transl Med. 2013 Mar 13; 5 (176) : 176ra34.
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Erez A, Nagamani SC, Shchelochkov OA, Premkumar MH, Campeau PM, Chen Y, Garg HK, Li L, Mian A, Bertin TK, Black JO, Zeng H, Tang Y, Reddy AK, Summar M, O'Brien WE, Harrison DG, Mitch WE, Marini JC, Aschner JL, Bryan NS, Lee B. " " Nat. Med.. 2011 Nov ; 17 (12) : 1619-26.
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Yang T, Mendoza-Londono R, Lu H, Tao J, Li K, Keller B, Jiang MM, Shah R, Chen Y, Bertin TK, Engin F, Dabovic B, Rifkin DB, Hicks J, Jamrich M, Beaudet AL, Lee B. " " J. Clin. Invest.. 2010 Jul 1; 120 (7) : 2474-85.
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Engin F, Yao Z, Yang T, Zhou G, Bertin T, Jiang MM, Chen Y, Wang L, Zheng H, Sutton RE, Boyce BF, Lee B. " " Nat. Med.. 2008 Mar ; 14 (3) : 299-305.
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Zhou G, Zheng Q, Engin F, Munivez E, Chen Y, Sebald E, Krakow D, Lee B. " " Proc. Natl. Acad. Sci. U.S.A.. 2006 Dec 12; 103 (50) : 19004-9.
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Morello R, Bertin TK, Chen Y, Hicks J, Tonachini L, Monticone M, Castagnola P, Rauch F, Glorieux FH, Vranka J, Bächinger HP, Pace JM, Schwarze U, Byers PH, Weis M, Fernandes RJ, Eyre DR, Yao Z, Boyce BF, Lee B. " " Cell. 2006 Oct 20; 127 (2) : 291-304.
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Toietta G, Mane VP, Norona WS, Finegold MJ, Ng P, McDonagh AF, Beaudet AL, Lee B. " " Proc. Natl. Acad. Sci. U.S.A.. 2005 Mar 15; 102 (11) : 3930-5.
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Lee B, Yu H, Jahoor F, O'Brien W, Beaudet AL, Reeds P. " " Proc. Natl. Acad. Sci. U S A. 2000 Jul 5; 97 (14) : 8021-6.
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Morello R, Zhou G, Dreyer SD, Harvey SJ, Ninomiya Y, Thorner PS, Miner JH, Cole W, Winterpacht A, Zabel B, Oberg KC, Lee B. " " Nat Genet.. 2001 Feb ; 27 (2) : 205-8.
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Memberships

American Society of Human Genetics: Member

Society for Pediatric Research: Member

American College of Medical Genetics: Member

American Society for Clinical Investigation: Member

Society for Inherited Metabolic Disease: Member

National Academy of Medicine

Association of American Physicians

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