Johnson M. Liu, MD

Associate Professor, Pediatric Hematology & Oncology, The Karches Center for Oncology Research, 
The Feinstein Institute for Medical Research

Professor of Pediatrics, Hofstra Northwell School of Medicine

Phone: (516) 562-4844

About the Investigator

Dr. Johnson Liu received his medical degree from the University of Michigan Medical School. After residency training in internal medicine, he joined the National Heart, Lung and Blood Institute in 1988, first as a clinical associate, hematology fellow, and then as tenure-track investigator in the hematology branch.

While at the National Institutes of Health (NIH), he was awarded a Commendation Medal from the United States Public Health Service. To date, Dr. Liu has authored or co-authored 103 papers, reviews, monographs and book chapters in the scientific and medical literature, and was named to the Les Nelkin Memorial Endowed Chair in Pediatric Oncology in 2005. He is currently a professor of pediatrics at the Hofstra Northwell School of Medicine.

Research Focus

Dr. Liu’s research career has focused on the molecular basis of hematopoietic stem cell disorders that are collectively referred to as the inherited bone marrow failure syndromes, and he has been actively engaged in translational research studies since his time at the NIH. There, he was responsible for the development of the first clinical protocol aimed at gene therapy for Fanconi anemia, guiding this project from the initial bench experiments in 1994 through the manufacturing and regulatory process to completion in 2000.

Dr. Liu’s current bench research at the Les Nelkin Memorial Pediatric Oncology Laboratory is aimed at understanding the molecular pathophysiology of Diamond Blackfan anemia and Shwachman-Diamond syndrome, two of the most commonly seen inherited bone marrow failure syndromes. His laboratory is also actively engaged in research on erythropoiesis (the process of blood production in the bone marrow) and sickle cell anemia. His clinical interests are in the management of benign and malignant hematologic disorders, with a particular focus on bone marrow failure and myelodysplastic syndromes.

Lionel Blanc, PhD

Lab Members

Abena Appiah-Kubi, MD


University of Michigan, Ann Arbor, MI
Degree: BS
Field of Study: Biomedical Sciences

University of Michigan, Ann Arbor, MI
Degree: MD
Field of Study: Medicine

Medical University of South Carolina, Charleston
Degree: Resident
Field of Study: Internal Medicine

National Heart, Lung, and Blood Institute, NIH
Degree: Clin. Assoc.
Field of Study: Internal Medicine

National Heart, Lung, and Blood Institute, NIH
Degree: Fellow
Field of Study: Hematology

Awards & Honors

1992-1996 Research Associate, Hematology Branch, NHLBI, Bethesda, MD
1996-2002 Tenure-Track Investigator, Hematology Branch, NHLBI, Bethesda, MD
1992-2002 Attending Physician, Hematology Branch, NHLBI, Bethesda, MD
2002-2005 Associate Professor of Medicine, Mount Sinai School of Medicine, New York, NY
2005-2011 Associate Professor of Pediatrics, Albert Einstein College of Medicine, Bronx, NY
2005-present Associate Investigator, The Feinstein Institute for Medical Research, Manhasset, NY Attending Physician, Cohen Children’s Medical Center of NY, New Hyde Park, NY
2011-present Professor of Pediatrics; Hofstra Northwell School of Medicine, Hempstead, NY

2001 Commendation Medal, US Public Health Service
2005 Les Nelkin Endowed Chair in Pediatric Oncology

  1. Yamamoto K, Nihrane A, Aglipay J, Sironi JJ, Arkin S, Lipton JM, Ouchi T, Liu JM: “Upregulated ATM gene expression and activated DNA crosslink-induced damage response checkpoint in Fanconi anemia: implications for carcinogenesis.” Mol Med 14:167-174, 2008.
  2. Nihrane A, Sezgin G, Dsilva S, Dellorusso P, Yamamoto K, Ellis SR, Liu JM: “Depletion of the Shwachman-Diamond syndrome gene product, SBDS, leads to growth inhibition and increased expression of OPG and VEGF-A.” Blood Cells Mol Dis 42:85-91, 2009
  3. Moore IV JB, Farrar JE, Arceci RJ, Liu JM, Ellis SR: “Distinct ribosome maturation defects in yeast models of Diamond Blackfan anemia and Shwachman Diamond syndrome.” Haematologica 95:57-64, 2010.
  4. Vaiselbuh SR, Edelman M, Lipton JM, Liu JM: “Ectopic human mesenchymal stem cell-coated scaffolds in NOD/SCID mice: an in vivo model of the leukemia niche.” Tissue Eng Part C Methods 16:1523-1531, 2010.
  5. Burwick N, Shimamura A, Liu JM: “Non-DBA disorders of ribosome function: Shwachman-Diamond syndrome and 5q- syndrome.” Semin Hematol 48:136-143, 2011.
  6. Dror Y, Donadieu J, Koglmeier J, Dodge J, Toiviainen-Salo S, Makitie O, Kerr E, Zeidler C, Shimamura A, Shah N, Cipolli M, Kuijpers T, Durie P, Rommens J, Siderius L, Liu JM: “Draft consensus guidelines for diagnosis and treatment of Shwachman-Diamond syndrome.” Ann NY Acad Sci, 1242(1):40-55, 2011.
  7. Liu JM, Lipton JM, Mani S: “Sixth International Congress on Shwachman-Diamond syndrome: from patients to genes and back.” Ann NY Acad Sci, 1242(1):26-39, 2011.
  8. Sezgin G*, Henson AL*, Nihrane A, Singh SA, Wattenberg M, Alard P, Ellis SR, Liu JM: “Impaired growth, hematopoietic colony formation, and ribosome maturation in human cells depleted of Shwachman-Diamond syndrome protein SBDS.” Pediatr Blood Cancer 60:281-286, 2013.
  9. Liu JM, Lipton JM, Ellis SR: “Genetics of the ribosomopathies.” eLS (Wiley), in press 2013.

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