Scholars

Senior Investigator

Positions:

Senior Investigator, Molecular Hematopoiesis Section

Research Areas:

Research & Experimental Medicine, Cell Biology, Genetics & Heredity, General & Internal Medicine, Hematology, Biotechnology & Applied Microbiology, Science & Technology - Other Topics, Transplantation, Oncology, Biochemistry & Molecular Biology, Immunology, Dentistry, Oral Surgery & Medicine

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overview

Cynthia Dunbar earned a B.A. magna cum laude in the history of science from Harvard University in 1980 and a M.D. magna cum laude from Harvard Medical School in 1984. She subsequently completed her internal medicine internship and residency at Boston City Hospital and hematology fellowship training at the University of California, San Francisco. She came to the NHLBI as a postdoctoral fellow in the laboratory of Arthur Nienhuis in 1987, became an Investigator in 1993, and has been Head of the Molecular Hematopoiesis Section since 2000. Dr. Dunbar has received numerous awards for teaching, mentorship, and research, including the NIH Clinical Center Distinguished Clinical Teacher Award, the John Decker Memorial Lectureship, and the Brigham and Women's Hospital Moloney Award and Lectureship. She has authored more than 200 peer-reviewed scientific and review articles, and has given numerous invited lectures and presentations about her work. Dr. Dunbar is the Editor-in-Chief of the journal Blood, the flagship publication of the American Society of Hematology. She is also a member of the American Society for Clinical Investigation, a Master of the American College of Physicians, a founder and board member of the American Society for Cell and Gene Therapy, and served seventeen years as the director of the NIH Hematology Clinical Fellowship training program.

selected publications

Academic Articles248

(2020). Clonal tracking of erythropoiesis in rhesus macaques. HAEMATOLOGICA. 105(7), 1813-1824.
(2020). Immunosuppression and growth factors for severe aplastic anemia: new data for old questions. HAEMATOLOGICA. 105(5), 1170-1171.
(2019). Aberrant Clonal Hematopoiesis following Lentiviral Vector Transduction of HSPCs in a Rhesus Macaque. MOLECULAR THERAPY. 27(6), 1074-1086.
(2019). Busulfan Combined with Immunosuppression Allows Efficient Engraftment of Gene-Modified Cells in a Rhesus Macaque Model. MOLECULAR THERAPY. 27(9), 1586-1596.
(2019). CRISPR/Cas9 PIG-A gene editing in nonhuman primate model demonstrates no intrinsic clonal expansion of PNH HSPCs. BLOOD. 133(23), 2542-2545.
(2019). Eltrombopag maintains human hematopoietic stem and progenitor cells under inflammatory conditions mediated by IFN-gamma. BLOOD. 133(19), 2043-2055.
(2019). Impact of CMV Infection on Natural Killer Cell Clonal Repertoire in CMV-Naive Rhesus Macaques. Frontiers in Immunology. 10,
(2019). Telomere dynamics and hematopoietic differentiation of human DKC1-mutant induced pluripotent stem cells. Stem Cell Research. 40,
(2019). Treatment optimization and genomic outcomes in refractory severe aplastic anemia treated with eltrombopag. BLOOD. 133(24), 2575-2585.
(2018). An Introduction to the Analysis of Single-Cell RNA-Sequencing Data. Molecular Therapy-Methods & Clinical Development.
(2018). Barcoding of Macaque Hematopoietic Stem and Progenitor Cells: A Robust Platform to Assess Vector Genotoxicity. Molecular Therapy-Methods & Clinical Development. 11, 143-154.
(2018). Bone Marrow as a Source of Cells for Paroxysmal Nocturnal Hemoglobinuria Detection. AMERICAN JOURNAL OF CLINICAL PATHOLOGY. 150(3), 273-282.
(2018). Clonal expansion and compartmentalized maintenance of rhesus macaque NK cell subsets. Science Immunology. 3(29),
(2018). Dexpramipexole as an oral steroid-sparing agent in hypereosinophilic syndromes. BLOOD. 132(5), 501-509.
(2018). Efficient differentiation of cardiomyocytes and generation of calcium-sensor reporter lines from nonhuman primate iPSCs. Scientific Reports. 8,
(2018). Eltrombopag mobilizes iron in patients with aplastic anemia. BLOOD. 131(21), 2399-2402.
(2018). GATA2 deficiency and human hematopoietic development modeled using induced pluripotent stem cells. Blood Advances. 2(23), 3553-3565.
(2018). Genetic Inactivation of CD33 in Hematopoietic Stem Cells to Enable CAR T Cell Immunotherapy for Acute Myeloid Leukemia. CELL. 173(6), 1439-+.
(2018). Genotoxic Lemons Become Epigenomic Lemonade. Cell Stem Cell. 23(1), 9-10.
(2018). Geographic clonal tracking in macaques provides insights into HSPC migration and differentiation. JOURNAL OF EXPERIMENTAL MEDICINE. 215(1), 217-232.
(2018). Persistent elevation of plasma thrombopoietin levels after treatment in severe aplastic anemia. EXPERIMENTAL HEMATOLOGY. 58, 39-43.
(2018). The impact of aging on primate hematopoiesis as interrogated by clonal tracking. BLOOD. 131(11), 1195-1205.
(2017). Acquired somatic mutations in PNH reveal long-term maintenance of adaptive NK cells independent of HSPCs. BLOOD. 129(14), 1940-1946.
(2017). Adaptive NK cells can persist in patients with GATA2 mutation depleted of stem and progenitor cells. BLOOD. 129(14), 1927-1939.
(2017). Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia. NEW ENGLAND JOURNAL OF MEDICINE. 376(16), 1540-1550.
(2017). Quantitative stability of hematopoietic stem and progenitor cell clonal output in rhesus macaques receiving transplants. BLOOD. 129(11), 1448-1457.
(2017). Rhesus iPSC Safe Harbor Gene-Editing Platform for Stable Expression of Transgenes in Differentiated Cells of All Germ Layers. MOLECULAR THERAPY. 25(1), 44-53.
(2017). Transcriptome analysis reveals similarities between human blood CD3(-) CD56(bright) cells and mouse CD127(+) innate lymphoid cells. Scientific Reports. 7,
(2017). Two Decades of ASGCT: Dreams Become Reality. MOLECULAR THERAPY. 25(5), 1057-1058.
(2017). iPSCs and fibroblast subclones from the same fibroblast population contain comparable levels of sequence variations. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA. 114(8), 1964-1969.
(2016). Blood's 70th anniversary: CARs on the Blood highway. BLOOD. 128(1), 1-3.
(2016). Gene Editing of Human Hematopoietic Stem and Progenitor Cells: Promise and Potential Hurdles. HUMAN GENE THERAPY. 27(10), 729-740.
(2016). Gene and Cell Therapies in Expansion Mode: ASGCT 2016. MOLECULAR THERAPY. 24(8), 1333-1334.
(2016). The Role of Nonhuman Primate Animal Models in the Clinical Development of Pluripotent Stem Cell Therapies. MOLECULAR THERAPY. 24(7), 1165-1169.
(2016). Thrombopoietic status of patients on haemodialysis. BRITISH JOURNAL OF HAEMATOLOGY. 172(6), 954-957.
(2015). An AAVS1-Targeted Minigene Platform for Correction of iPSCs From All Five Types of Chronic Granulomatous Disease. MOLECULAR THERAPY. 23(1), 147-157.
(2015). Bone marrow skeletal stem/progenitor cell defects in dyskeratosis congenita and telomere biology disorders. BLOOD. 125(5), 793-802.
(2015). Eltrombopag in Aplastic Anemia. SEMINARS IN HEMATOLOGY. 52(1), 31-37.
(2015). Functional Niche Competition Between Normal Hematopoietic Stem and Progenitor Cells and Myeloid Leukemia Cells. STEM CELLS. 33(12), 3635-3642.
(2015). Human hematopoietic stem cells from mobilized peripheral blood can be purified based on CD49f integrin expression. BLOOD. 126(13), 1631-1633.
(2015). Modeling Human Bone Marrow Failure Syndromes Using Pluripotent Stem Cells and Genome Engineering. MOLECULAR THERAPY. 23(12), 1832-1842.
(2015). Regulated Apoptosis of Genetically Modified Hematopoietic Stem and Progenitor Cells Via an Inducible Caspase-9 Suicide Gene in Rhesus Macaques. STEM CELLS. 33(1), 91-100.
(2014). Angioedema in patients treated with sirolimus and ACE inhibitor post hematopoietic SCT. BONE MARROW TRANSPLANTATION. 49(11), 1448-1449.
(2014). Clonal Tracking of Rhesus Macaque Hematopoiesis Highlights a Distinct Lineage Origin for Natural Killer Cells. Cell Stem Cell. 14(4), 486-499.
(2014). Development of an inducible caspase-9 safety switch for pluripotent stem cell-based therapies. Molecular Therapy-Methods & Clinical Development. 1,
(2014). Dynamics of HSPC Repopulation in Nonhuman Primates Revealed by a Decade-Long Clonal-Tracking Study. Cell Stem Cell. 14(4), 473-485.
(2014). Eltrombopag restores trilineage hematopoiesis in refractory severe aplastic anemia that can be sustained on discontinuation of drug. BLOOD. 123(12), 1818-1825.
(2014). IFN-gamma regulates survival and function of tumor-induced CD11b(+)Gr-1(high) myeloid derived suppressor cells by modulating the anti-apoptotic molecule Bcl2a1. EUROPEAN JOURNAL OF IMMUNOLOGY. 44(8), 2457-2467.
(2014). In vivo Clonal Tracking of Hematopoietic Stem and Progenitor Cells Marked by Five Fluorescent Proteins using Confocal and Multiphoton Microscopy. Jove-Journal of Visualized Experiments. (90),
(2014). No Impact of Lentiviral Transduction on Hematopoietic Stem/Progenitor Cell Telomere Length or Gene Expression in the Rhesus Macaque Model. MOLECULAR THERAPY. 22(1), 52-58.
(2014). Path to the Clinic: Assessment of iPSC-Based Cell Therapies In Vivo in a Nonhuman Primate Model. Cell Reports. 7(4), 1298-1309.
(2014). Patients with myeloid malignancies bearing PDGFRB fusion genes achieve durable long-term remissions with imatinib. BLOOD. 123(23), 3574-3577.
(2013). Active thrombopoiesis is associated with worse severity and activity of chronic GVHD. BONE MARROW TRANSPLANTATION. 48(12), 1569-1573.
(2013). Assessing the Risks of Genotoxicity in the Therapeutic Development of Induced Pluripotent Stem Cells. MOLECULAR THERAPY. 21(2), 272-281.
(2013). Defective telomere elongation and hematopoiesis from telomerase-mutant aplastic anemia iPSCs. JOURNAL OF CLINICAL INVESTIGATION. 123(5), 1952-1963.
(2013). Differences in the Phenotype, Cytokine Gene Expression Profiles, and In Vivo Alloreactivity of T Cells Mobilized with Plerixafor Compared with G-CSF. JOURNAL OF IMMUNOLOGY. 191(12), 6241-6249.
(2013). Hematopoietic Stem Cell Gene Therapy: Assessing the Relevance of Preclinical Models. SEMINARS IN HEMATOLOGY. 50(2), 101-130.
(2013). High Efficiency Restriction Enzyme-Free Linear Amplification-Mediated Polymerase Chain Reaction Approach for Tracking Lentiviral Integration Sites Does Not Abrogate Retrieval Bias. HUMAN GENE THERAPY. 24(1), 38-47.
(2013). Integration-specific In Vitro Evaluation of Lentivirally Transduced Rhesus CD34(+) Cells Correlates With In Vivo Vector Copy Number. Molecular Therapy-Nucleic Acids. 2,
(2013). Measurement of the absolute immature platelet number reflects marrow production and is not impacted by platelet transfusion. TRANSFUSION. 53(6), 1201-1204.
(2013). Pathophysiology and management of thrombocytopenia in bone marrow failure: possible clinical applications of TPO receptor agonists in aplastic anemia and myelodysplastic syndromes. INTERNATIONAL JOURNAL OF HEMATOLOGY. 98(1), 48-55.
(2012). An educational infusion: more synergy between Blood and the American Society of Hematology. BLOOD. 120(23), 4449-4449.
(2012). BCL2A1a Over-Expression in Murine Hematopoietic Stem and Progenitor Cells Decreases Apoptosis and Results in Hematopoietic Transformation. PLoS One. 7(10),
(2012). Bone marrow homing and engraftment of human hematopoietic stem and progenitor cells is mediated by a polarized membrane domain. BLOOD. 119(8), 1848-1855.
(2012). Challenges in Vector and Trial Design Using Retroviral Vectors for Long-Term Gene Correction in Hematopoietic Stem Cell Gene Therapy. MOLECULAR THERAPY. 20(6), 1084-1094.
(2012). Dynamic clonal analysis of murine hematopoietic stem and progenitor cells marked by 5 fluorescent proteins using confocal and multiphoton microscopy. BLOOD. 120(26), E105-E116.
(2012). Eltrombopag and Improved Hematopoiesis in Refractory Aplastic Anemia. NEW ENGLAND JOURNAL OF MEDICINE. 367(1), 11-19.
(2012). Hematopoietic stem cell engineering at a crossroads. BLOOD. 119(5), 1107-1116.
(2012). Pharmacological Modulation of Humoral Immunity in a Nonhuman Primate Model of AAV Gene Transfer for Hemophilia B. MOLECULAR THERAPY. 20(7), 1410-1416.
(2012). Thymidine Kinase Suicide Gene-mediated Ganciclovir Ablation of Autologous Gene-modified Rhesus Hematopoiesis. MOLECULAR THERAPY. 20(10), 1932-1943.
(2012). Transient silencing of PTEN in human CD34(+) cells enhances their proliferative potential and ability to engraft immunodeficient mice. EXPERIMENTAL HEMATOLOGY. 40(1), 84-91.
(2011). CD9 up-regulation on CD34(+) cells with ingenol 3,20-dibenzoate does not improve homing in NSG mice. BLOOD. 117(21), 5774-5776.
(2011). Contributions of Gene Marking to Cell and Gene Therapies. HUMAN GENE THERAPY. 22(6), 659-668.
(2011). Human and rhesus macaque hematopoietic stem cells cannot be purified based only on SLAM family markers. BLOOD. 117(5), 1550-1554.
(2011). Insertion Sites in Engrafted Cells Cluster Within a Limited Repertoire of Genomic Areas After Gammaretroviral Vector Gene Therapy. MOLECULAR THERAPY. 19(11), 2031-2039.
(2011). Patients, hematologists, and time. BLOOD. 117(10), 2753-2754.
(2011). Rapid mobilization of hematopoietic progenitors by AMD3100 and catecholamines is mediated by CXCR4-dependent SDF-1 release from bone marrow stromal cells. LEUKEMIA. 25(8), 1286-1296.
(2011). Sox4 cooperates with PU.1 haploinsufficiency in murine myeloid leukemia. BLOOD. 118(17), 4674-4681.
(2011). Stem cell gene therapy: the risks of insertional mutagenesis and approaches to minimize genotoxicity..
(2010). AAV5-mediated gene transfer to the parotid glands of non-human primates. GENE THERAPY. 17(1), 50-60.
(2010). Evaluation of a rapamycin-regulated serotype 2 adeno-associated viral vector in macaque parotid glands. ORAL DISEASES. 16(3), 269-277.
(2010). Ex Vivo Expansion of Retrovirally Transduced Primate CD34+Cells Results in Overrepresentation of Clones With MDS1/EVI1 Insertion Sites in the Myeloid Lineage After Transplantation. MOLECULAR THERAPY. 18(9), 1633-1639.
(2010). Feline leukemia virus integrase and capsid packaging functions do not change the insertion profile of standard Moloney retroviral vectors. GENE THERAPY. 17(6), 799-804.
(2010). Gene therapy activates EVI1, destabilizes chromosomes. NATURE MEDICINE. 16(2), 163-165.
(2010). Graft-versus-Host Disease: Role of Inflammation in the Development of Chromosomal Abnormalities of Keratinocytes. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION. 16(12), 1665-1673.
(2010). Keeping up with Blood: introducing our CME program. BLOOD. 115(4), 756-756.
(2010). Molecular characterisation of side population cells with cancer stem cell-like characteristics in small-cell lung cancer. BRITISH JOURNAL OF CANCER. 102(11), 1636-1644.
(2010). No Evidence for Clonal Selection Due to Lentiviral Integration Sites in Human Induced Pluripotent Stem Cells. STEM CELLS. 28(4), 687-694.
(2010). Repetitive Busulfan Administration After Hematopoietic Stem Cell Gene Therapy Associated with a Dominant HDAC7 Clone in a Nonhuman Primate. HUMAN GENE THERAPY. 21(6), 695-703.
(2009). "Ghostbusting" at Blood. BLOOD. 113(3), 502-503.
(2009). Genetically Modified CD34(+) Hematopoietic Stem Cells Contribute to Turnover of Brain Perivascular Macrophages in Long-Term Repopulated Primates. AMERICAN JOURNAL OF PATHOLOGY. 174(5), 1808-1817.
(2009). In vivo selection of hematopoietic progenitor cells and temozolomide dose intensification in rhesus macaques through lentiviral transduction with a drug resistance gene. JOURNAL OF CLINICAL INVESTIGATION. 119(7), 1952-1963.
(2009). Intercellular transfer to signalling endosomes regulates an ex vivo bone marrow niche. NATURE CELL BIOLOGY. 11(3), 303-U168.
(2009). Introducing "e-Blood". BLOOD. 113(19), 4488-4488.
(2009). More frequent Blood transfusions. BLOOD. 113(1), 6-6.
(2009). Sustained high-level polyclonal hematopoietic marking and transgene expression 4 years after autologous transplantation of rhesus macaques with SIV lentiviral vector-transduced CD34(+) cells. BLOOD. 113(22), 5434-5443.
(2008). Donor demographic and laboratory predictors of allogeneic peripheral blood stem cell mobilization in an ethnically diverse population. BLOOD. 112(5), 2092-2100.
(2008). HOXB4 and retroviral vectors: adding fuel to the fire. JOURNAL OF CLINICAL INVESTIGATION. 118(4), 1350-1353.
(2008). Reduced genotoxicity of avian sarcoma leukosis virus vectors in rhesus long-term repopulating cells compared to standard murine retrovirus vectors. MOLECULAR THERAPY. 16(9), 1617-1623.
(2008). Sorting of Transgenic Secretory Proteins in Rhesus Macaque Parotid Glands After Adenovirus-Mediated Gene Transfer. HUMAN GENE THERAPY. 19(12), 1401-1405.

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Academic Articles248