Neal Young, M.D.

Senior Investigator

Hematopoiesis and Bone Marrow Failure


10 Center Dr
Bethesda, MD 20814
United States


Research Topics

Research in the Cell Biology Section spans the basic sciences, clinical trials, and epidemiology. Bench work involves methods of cell and molecular biology, immunology, and virology. Blood cell production in healthy individuals and especially in patients with bone marrow failure is the main theme. Advanced techniques most recently include single cell RNAseq, CRISPR-Cas9 gene editing, multicolored bar coded flow cytometry, and SomaLogic deep proteomics. Use of patient samples is encouraged, as the Hematology Branch clinic for patients with aplastic anemia, paroxysmal nocturnal hemoglobinuria (PNH), myelodysplastic syndromes (MDS), large granular lymphocytosis (LGL), and constitutional marrow failure is one of the largest in the world. Results in the laboratory have had real impact on the care of patients with these life-threatening illnesses, and access to patient samples over time and with therapeutic perturbation makes the laboratory strategies both innovative and concrete.

Some Current Laboratory Projects

Single cell RNAseq of normal and diseased bone marrow stem and progenitor cells

Gene editing in human cell lines of genes that affect the epigenome

RNAseq and gene editing applied to PNH cells

Murine models of immune mediated marrow failure, potential therapies and mechanisms

Sex hormone action on human marrow cells

Some Current and Planned Clinical Protocals

Eltrombopag in marrow failure, including treatment-naïve SAA, MAA, and MDS

Campath in LGL

Rapamycin to prevent relapse in AA

Sex hormone modulation of telomeres in patients with telomeropathies

Organization and Mentoring

The Cell Biology Section is large and has a nucleus of expert senior staff to provide training and supervision in a wide variety of advanced techniques. The Section is part of the Hematology Branch, and there is close interaction among all the Branch’s members, including sharing of resources and expertise. There is an active program of lectures and group meetings. In addition to knowledge acquisition, clear thinking and crisp verbal and written expression are priorities for development in trainees.

Dr. Young’s former trainees are leaders in hematology in the United States, Asia, Europe, and South America.


Neal Young received a A.B. cum laude from Harvard College in 1967 and his M.D. in 1971 from the Johns Hopkins School of Medicine. He did post-graduate medical training at Massachusetts General Hospital and Barnes Hospital at Washington University in St. Louis before joining the NHLBI in 1981. He is a Master of the American College of Physicians and has accrued many academic honors. His efforts as a US Government employee were recognized with Samuel J. Heyman Service to America Medal in Science and the Environment. Dr. Young has authored over 280 original scientific and medical articles in peer reviewed journals and more than 160 reviews and book chapters. He also is author or editor of 10 medical and scientific books, including a new textbook of hematology. Dr. Young is a member of the American Society for Clinical Investigation, American Association of Physicians, American Federation for Clinical Research, American Society of Hematology, and International Society for Experimental Hematology. He is co-inventor on 7 patents concerning B19 parvovirus. Dr. Young’s trainees head departments of hematology worldwide. He has lectured and published prolifically in hematology. He led a novel training program in Vietnam that transformed that country’s approach to blood diseases. He now heads a successful new trans-NIH initiative in human immunology.

Selected Publications

  1. Mustjoki S, Young NS. Somatic Mutations in "Benign" Disease. N Engl J Med. 2021;384(21):2039-2052.
  2. Young NS. Aplastic Anemia. N Engl J Med. 2018;379(17):1643-1656.
  3. Townsley DM, Scheinberg P, Winkler T, Desmond R, Dumitriu B, Rios O, Weinstein B, Valdez J, Lotter J, Feng X, Desierto M, Leuva H, Bevans M, Wu C, Larochelle A, Calvo KR, Dunbar CE, Young NS. Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia. N Engl J Med. 2017;376(16):1540-1550.
  4. Gutierrez-Rodrigues F, Munger E, Ma X, Groarke EM, Tang Y, Patel BA, Catto LFB, Clé DV, Niewisch MR, Alves-Paiva RM, Donaires FS, Pinto AL, Borges G, Santana BA, McReynolds LJ, Giri N, Altintas B, Fan X, Shalhoub R, Siwy CM, Diamond C, Raffo DQ, Craft K, Kajigaya S, Summers RM, Liu P, Cunningham L, Hickstein DD, Dunbar CE, Pasquini R, De Oliveira MM, Velloso EDRP, Alter BP, Savage SA, Bonfim C, Wu CO, Calado RT, Young NS. Differential diagnosis of bone marrow failure syndromes guided by machine learning. Blood. 2023;141(17):2100-2113.
  5. Yoshizato T, Dumitriu B, Hosokawa K, Makishima H, Yoshida K, Townsley D, Sato-Otsubo A, Sato Y, Liu D, Suzuki H, Wu CO, Shiraishi Y, Clemente MJ, Kataoka K, Shiozawa Y, Okuno Y, Chiba K, Tanaka H, Nagata Y, Katagiri T, Kon A, Sanada M, Scheinberg P, Miyano S, Maciejewski JP, Nakao S, Young NS, Ogawa S. Somatic Mutations and Clonal Hematopoiesis in Aplastic Anemia. N Engl J Med. 2015;373(1):35-47.

Related Scientific Focus Areas

This page was last updated on Tuesday, April 23, 2024