The goal of the Orkin laboratory is to understand how commitment to specific blood lineages is programmed and how cell-specific patterns of gene expression are established. Since gene expression is controlled by nuclear regulatory factors (transcription factors), efforts have centered on identifying those crucial for development of stem cells or individual lineages. Research encompasses conventional molecular biology and contemporary mouse genetics in three focus areas:

  • Lineage specification and hematopoietic differentiation
    Red blood cells and megakaryocytes (which produce platelets) share a common precursor cell. They discovered a transcription factor, GATA-1, that participates in the regulation of virtually all red cell (erythroid) and megakaryocyte-specific expressed genes. Expression of GATA-1 in progenitors drives cells toward erythroid and megakaryocytic fates. Disruption of the GATA-1 gene in mice leads to a failure of maturation of both lineages. In addition to controlling end-stage markers of these lineages, GATA-1 influences proliferation and cell death decisions of precursor cells. Using this mouse model to dissect mechanisms of cell differentiation, Orkin and colleagues have sought to understand how GATA-1 functions in transcription. This line of investigation led us to hypothesize and then discover a cofactor required for GATA-1's function in these lineages. This novel cofactor, FOG (for friend of GATA-1), is also essential for normal red blood cell and megakaryocyte development. Current research focuses on how FOG modulates the function of GATA-1, how others factors (eg Gfi-1b) cooperate in differentiation, and the discovery of protein partners of these key regulators of development.

  • Hematopoiesis-leukemia interface
    Remarkably, many genes essential for normal hematopoietic development are involved in chromosomal translocations in human leukemias. This finding reflects perturbation of normal homeostasis by expression of an altered protein or excessive production of an otherwise normal factor. The Orkin lab is studying several transcription factors (e.g. SCL/tal-1, TEL, and GATA-1) affected by genetic events in leukemias. Of particular interest are infant leukemia of the megakaryoblastic variety occurring in the setting of Down syndrome and somatic mutation of GATA-1 and childhood TEL-AML B-cell ALL.

  • Modeling the first steps in human cancer in the mouse
    In an effort to improve models of human cancers the laboratory is generating gene targeted mice with conditional mutations that replicate the first steps in selected human cancers. For example, mice have been created that conditionally express the TEL-Ntrk fusion gene characteristic of one form of breast cancer. These mice develop invasive breast cancer with full penetrance and a consistent time-course. These mice are being used to explore the step-wise pathogenesis of breast cancer and the potential role of cancer stem cells.

  • Stem cell biology
    Stem cells provide for production of specific cell lineages during development and throughout adult life. Dr. Orkin and colleagues are focusing efforts on characterization of the genes required for hematopoietic stem self-renewal and survival, as well as genes within embryonic stem cells that maintain pluripotency. Characterization of the molecular underpinnings of stem cell functions will have broad implications for cellular development and cancer.


Dr. Orkin received his MD in 1972 from Harvard Medical School, followed by postdoctoral research at the National Institutes of Health and clinical training in pediatrics and hematology-oncology at Children's Hospital Boston and DFCI, where he joined the faculty in 1978. Dr. Orkin is a Howard Hughes Medical Institute Investigator and was elected to the 2019-2020 Council of the National Academy of Medicine. Over the past decade, his laboratory has defined critical nuclear regulators of hematopoiesis.


  • Harrington Prize for Innovation in Medicine, American Society for Clinical Investigation and Harrington Discovery Institute, 2020
  • King Faisal International Prize for Medicine, King Faisal Foundation, 2020
  • Mechthild Esser Nemmers Prize in Medical Science, Northwestern University, 2018
  • George M. Kober Medal, American Association of Physicians, 2018
  • Clotten Foundation Prize, 2017
  • Member, American Philosophical Society, 2017
  • Lifetime Impact award, Boston Children’s Hospital’s Global Pediatric Innovation Summit, 2015
  • William A. Allan Award, American Society for Human Genetics, 2014
  • Jessie Stevenson Kovalenko Medal, National Academy of Sciences, 2013
  • Donald Metcalf Award, International Society of Experimental Hematology, 2012
  • Basic Science Mentor Award, American Society of Hematology, 2009
  • Legends in Hematology, American Society of Hematology, 2008
  • AAMC Award for Distinguished Research in Medical Sciences, 2005
  • Frank Oski Award, American Society of Pediatric Hematology and Oncology, 2003
  • E. Donnell Thomas Prize, American Society of Hematology, 1998
  • Helmut Horten Research Award, 1995
  • Warren Alpert Foundation Prize, 1993
  • Member, American Academy of Arts and Sciences, 1993
  • Member, Institute of Medicine, 1992
  • Member, National Academy of Sciences, 1991
  • E. Mead Johnson Award, Society for Pediatric Research, 1987
  • William Dameshek Prize, American Society of Hematology, 1986
  • Research Career Development Award, National Institutes of Health, 1979
  • Young Investigator Award, National Institutes of Health, 1976

Press Releases


Publications powered by Harvard Catalyst Profiles

  1. Transcription factor-mediated intestinal metaplasia and the role of a shadow enhancer. Genes Dev. 2021 Dec 30. View abstract
  2. Inner nuclear protein Matrin-3 coordinates cell differentiation by stabilizing chromatin architecture. Nat Commun. 2021 10 29; 12(1):6241. View abstract
  3. Dietary suppression of MHC class II expression in intestinal epithelial cells enhances intestinal tumorigenesis. Cell Stem Cell. 2021 11 04; 28(11):1922-1935.e5. View abstract
  4. Mapping the evolving landscape of super-enhancers during cell differentiation. Genome Biol. 2021 09 15; 22(1):269. View abstract
  5. A unified model of human hemoglobin switching through single-cell genome editing. Nat Commun. 2021 08 17; 12(1):4991. View abstract
  6. Reactivation of a developmentally silenced embryonic globin gene. Nat Commun. 2021 07 21; 12(1):4439. View abstract
  7. Indispensable epigenetic control of thymic epithelial cell development and function by polycomb repressive complex 2. Nat Commun. 2021 06 24; 12(1):3933. View abstract
  8. Author Correction: Transcription factor competition at the ?-globin promoters controls hemoglobin switching. Nat Genet. 2021 Apr; 53(4):586. View abstract
  9. Transcription factor competition at the ?-globin promoters controls hemoglobin switching. Nat Genet. 2021 04; 53(4):511-520. View abstract
  10. MOLECULAR MEDICINE: Found in Translation. Med (N Y). 2021 Feb 12; 2(2):122-136. View abstract
  11. ARID4B is critical for mouse embryonic stem cell differentiation towards mesoderm and endoderm, linking epigenetics to pluripotency exit. J Biol Chem. 2020 12 18; 295(51):17738-17751. View abstract
  12. Arid4b is critical for mouse embryonic stem cell differentiation towards mesoderm and endoderm, linking epigenetics to pluripotency exit. J Biol Chem. 2020 Oct 15. View abstract
  13. A saturating mutagenesis CRISPR-Cas9-mediated functional genomic screen identifies cis- and trans-regulatory elements of Oct4 in murine ESCs. J Biol Chem. 2020 11 20; 295(47):15797-15809. View abstract
  14. Enhancer dependence of cell-type-specific gene expression increases with developmental age. Proc Natl Acad Sci U S A. 2020 09 01; 117(35):21450-21458. View abstract
  15. An Engineered CRISPR-Cas9 Mouse Line for Simultaneous Readout of Lineage Histories and Gene Expression Profiles in Single Cells. Cell. 2020 Jun 25; 181(7):1693-1694. View abstract
  16. An Engineered CRISPR-Cas9 Mouse Line for Simultaneous Readout of Lineage Histories and Gene Expression Profiles in Single Cells. Cell. 2020 06 11; 181(6):1410-1422.e27. View abstract
  17. Multiplexed capture of spatial configuration and temporal dynamics of locus-specific 3D chromatin by biotinylated dCas9. Genome Biol. 2020 03 05; 21(1):59. View abstract
  18. Live-animal imaging of native haematopoietic stem and progenitor cells. Nature. 2020 02; 578(7794):278-283. View abstract
  19. Control of human hemoglobin switching by LIN28B-mediated regulation of BCL11A translation. Nat Genet. 2020 02; 52(2):138-145. View abstract
  20. CUT&RUNTools: a flexible pipeline for CUT&RUN processing and footprint analysis. Genome Biol. 2019 09 09; 20(1):192. View abstract
  21. BORIS promotes chromatin regulatory interactions in treatment-resistant cancer cells. Nature. 2019 08; 572(7771):676-680. View abstract
  22. Rational targeting of a NuRD subcomplex guided by comprehensive in situ mutagenesis. Nat Genet. 2019 07; 51(7):1149-1159. View abstract
  23. TAF5L and TAF6L Maintain Self-Renewal of Embryonic Stem Cells via the MYC Regulatory Network. Mol Cell. 2019 06 20; 74(6):1148-1163.e7. View abstract
  24. Extensive Recovery of Embryonic Enhancer and Gene Memory Stored in Hypomethylated Enhancer DNA. Mol Cell. 2019 05 02; 74(3):542-554.e5. View abstract
  25. Single-Cell Analysis Identifies LY6D as a Marker Linking Castration-Resistant Prostate Luminal Cells to Prostate Progenitors and Cancer. Cell Rep. 2018 12 18; 25(12):3504-3518.e6. View abstract
  26. Yap1 safeguards mouse embryonic stem cells from excessive apoptosis during differentiation. Elife. 2018 12 18; 7. View abstract
  27. CRISPR-SURF: discovering regulatory elements by deconvolution of CRISPR tiling screen data. Nat Methods. 2018 12; 15(12):992-993. View abstract
  28. PRC2 loss induces chemoresistance by repressing apoptosis in T cell acute lymphoblastic leukemia. J Exp Med. 2018 12 03; 215(12):3094-3114. View abstract
  29. FAM210B is an erythropoietin target and regulates erythroid heme synthesis by controlling mitochondrial iron import and ferrochelatase activity. J Biol Chem. 2018 12 21; 293(51):19797-19811. View abstract
  30. Emerging Genetic Therapy for Sickle Cell Disease. Annu Rev Med. 2019 01 27; 70:257-271. View abstract
  31. Polycomb Repressive Complex 2 is essential for development and maintenance of a functional TEC compartment. Sci Rep. 2018 09 25; 8(1):14335. View abstract
  32. Canonical PRC2 function is essential for mammary gland development and affects chromatin compaction in mammary organoids. PLoS Biol. 2018 08; 16(8):e2004986. View abstract
  33. Author Correction: High-fat diet enhances stemness and tumorigenicity of intestinal progenitors. Nature. 2018 08; 560(7717):E26. View abstract
  34. Downregulation of Endothelin Receptor B Contributes to Defective B Cell Lymphopoiesis in Trisomy 21 Pluripotent Stem Cells. Sci Rep. 2018 05 22; 8(1):8001. View abstract
  35. Mapping the Mouse Cell Atlas by Microwell-Seq. Cell. 2018 05 17; 173(5):1307. View abstract
  36. The Polycomb-Dependent Epigenome Controls ß Cell Dysfunction, Dedifferentiation, and Diabetes. Cell Metab. 2018 Jun 05; 27(6):1294-1308.e7. View abstract
  37. 14q32 and let-7 microRNAs regulate transcriptional networks in fetal and adult human erythroblasts. Hum Mol Genet. 2018 04 15; 27(8):1411-1420. View abstract
  38. Integrated design, execution, and analysis of arrayed and pooled CRISPR genome-editing experiments. Nat Protoc. 2018 05; 13(5):946-986. View abstract
  39. Direct Promoter Repression by BCL11A Controls the Fetal to Adult Hemoglobin Switch. Cell. 2018 04 05; 173(2):430-442.e17. View abstract
  40. Dissecting super-enhancer hierarchy based on chromatin interactions. Nat Commun. 2018 03 05; 9(1):943. View abstract
  41. Genome-wide CRISPR-Cas9 Screen Identifies Leukemia-Specific Dependence on a Pre-mRNA Metabolic Pathway Regulated by DCPS. Cancer Cell. 2018 03 12; 33(3):386-400.e5. View abstract
  42. Mapping the Mouse Cell Atlas by Microwell-Seq. Cell. 2018 02 22; 172(5):1091-1107.e17. View abstract
  43. Regulation of embryonic haematopoietic multipotency by EZH1. Nature. 2018 01 25; 553(7689):506-510. View abstract
  44. Human genetic variation alters CRISPR-Cas9 on- and off-targeting specificity at therapeutically implicated loci. Proc Natl Acad Sci U S A. 2017 12 26; 114(52):E11257-E11266. View abstract
  45. Recent progress in understanding and manipulating haemoglobin switching for the haemoglobinopathies. Br J Haematol. 2018 03; 180(5):630-643. View abstract
  46. First critical repressive H3K27me3 marks in embryonic stem cells identified using designed protein inhibitor. Proc Natl Acad Sci U S A. 2017 09 19; 114(38):10125-10130. View abstract
  47. PRMT1-Mediated Translation Regulation Is a Crucial Vulnerability of Cancer. Cancer Res. 2017 09 01; 77(17):4613-4625. View abstract
  48. Gene correction of HAX1 reversed Kostmann disease phenotype in patient-specific induced pluripotent stem cells. Blood Adv. 2017 Jun 13; 1(14):903-914. View abstract
  49. The 2017 ASPHO distinguished career award goes to Holcombe E. Grier, MD. Pediatr Blood Cancer. 2017 06; 64 Suppl 1. View abstract
  50. A molecular roadmap for induced multi-lineage trans-differentiation of fibroblasts by chemical combinations. Cell Res. 2017 06; 27(6):842. View abstract
  51. A molecular roadmap for induced multi-lineage trans-differentiation of fibroblasts by chemical combinations. Cell Res. 2017 06; 27(6):843. View abstract
  52. Erythropoietin signaling regulates heme biosynthesis. Elife. 2017 05 29; 6. View abstract
  53. Challenges and emerging directions in single-cell analysis. Genome Biol. 2017 05 08; 18(1):84. View abstract
  54. Reduced Erg Dosage Impairs Survival of Hematopoietic Stem and Progenitor Cells. Stem Cells. 2017 07; 35(7):1773-1785. View abstract
  55. EED orchestration of heart maturation through interaction with HDACs is H3K27me3-independent. Elife. 2017 04 10; 6. View abstract
  56. Functional interrogation of non-coding DNA through CRISPR genome editing. Methods. 2017 05 15; 121-122:118-129. View abstract
  57. Transcription control by the ENL YEATS domain in acute leukaemia. Nature. 2017 03 09; 543(7644):270-274. View abstract
  58. Variant-aware saturating mutagenesis using multiple Cas9 nucleases identifies regulatory elements at trait-associated loci. Nat Genet. 2017 Apr; 49(4):625-634. View abstract
  59. Characterization of genomic deletion efficiency mediated by clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 nuclease system in mammalian cells. J Biol Chem. 2017 02 10; 292(6):2556. View abstract
  60. A molecular roadmap for induced multi-lineage trans-differentiation of fibroblasts by chemical combinations. Cell Res. 2017 03; 27(3):386-401. View abstract
  61. The histone demethylase UTX regulates the lineage-specific epigenetic program of invariant natural killer T cells. Nat Immunol. 2017 02; 18(2):184-195. View abstract
  62. Strict in vivo specificity of the Bcl11a erythroid enhancer. Blood. 2016 11 10; 128(19):2338-2342. View abstract
  63. CellMapper: rapid and accurate inference of gene expression in difficult-to-isolate cell types. Genome Biol. 2016 09 29; 17(1):201. View abstract
  64. Bcl11a Deficiency Leads to Hematopoietic Stem Cell Defects with an Aging-like Phenotype. Cell Rep. 2016 09 20; 16(12):3181-3194. View abstract
  65. Chronic Myelogenous Leukemia- Initiating Cells Require Polycomb Group Protein EZH2. Cancer Discov. 2016 11; 6(11):1237-1247. View abstract
  66. Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype. J Clin Invest. 2016 10 03; 126(10):3868-3878. View abstract
  67. Single-Cell Transcript Profiles Reveal Multilineage Priming in Early Progenitors Derived from Lgr5(+) Intestinal Stem Cells. Cell Rep. 2016 08 23; 16(8):2053-2060. View abstract
  68. Mathematical modeling of erythrocyte chimerism informs genetic intervention strategies for sickle cell disease. Am J Hematol. 2016 09; 91(9):931-7. View abstract
  69. Analyzing CRISPR genome-editing experiments with CRISPResso. Nat Biotechnol. 2016 07 12; 34(7):695-7. View abstract
  70. Loss of Ezh2 synergizes with JAK2-V617F in initiating myeloproliferative neoplasms and promoting myelofibrosis. J Exp Med. 2016 07 25; 213(8):1479-96. View abstract
  71. The Public Repository of Xenografts Enables Discovery and Randomized Phase II-like Trials in Mice. Cancer Cell. 2016 07 11; 30(1):183. View abstract
  72. An Achilles' Heel for MLL-Rearranged Leukemias: Writers and Readers of H3 Lysine 36 Dimethylation. Cancer Discov. 2016 07; 6(7):700-2. View abstract
  73. Adenosine-to-inosine RNA editing by ADAR1 is essential for normal murine erythropoiesis. Exp Hematol. 2016 10; 44(10):947-63. View abstract
  74. Hemoglobin genetics: recent contributions of GWAS and gene editing. Hum Mol Genet. 2016 10 01; 25(R2):R99-R105. View abstract
  75. Polycomb repressive complex 2 regulates skeletal growth by suppressing Wnt and TGF-ß signalling. Nat Commun. 2016 06 22; 7:12047. View abstract
  76. MEDICINE. Paying for future success in gene therapy. Science. 2016 May 27; 352(6289):1059-61. View abstract
  77. Acquired Tissue-Specific Promoter Bivalency Is a Basis for PRC2 Necessity in Adult Cells. Cell. 2016 Jun 02; 165(6):1389-1400. View abstract
  78. Corrigendum: LSD1 is essential for oocyte meiotic progression by regulating CDC25B expression in mice. Nat Commun. 2016 Apr 20; 7:11467. View abstract
  79. Interferon-a signaling promotes embryonic HSC maturation. Blood. 2016 07 14; 128(2):204-16. View abstract
  80. The Public Repository of Xenografts Enables Discovery and Randomized Phase II-like Trials in Mice. Cancer Cell. 2016 04 11; 29(4):574-586. View abstract
  81. Customizing the genome as therapy for the ß-hemoglobinopathies. Blood. 2016 05 26; 127(21):2536-45. View abstract
  82. High-fat diet enhances stemness and tumorigenicity of intestinal progenitors. Nature. 2016 Mar 03; 531(7592):53-8. View abstract
  83. Ezh2 Controls an Early Hematopoietic Program and Growth and Survival Signaling in Early T Cell Precursor Acute Lymphoblastic Leukemia. Cell Rep. 2016 Mar 01; 14(8):1953-65. View abstract
  84. Recent advances in globin research using genome-wide association studies and gene editing. Ann N Y Acad Sci. 2016 03; 1368(1):5-10. View abstract
  85. Serum-Based Culture Conditions Provoke Gene Expression Variability in Mouse Embryonic Stem Cells as Revealed by Single-Cell Analysis. Cell Rep. 2016 Feb 02; 14(4):956-965. View abstract
  86. Transcription factors LRF and BCL11A independently repress expression of fetal hemoglobin. Science. 2016 Jan 15; 351(6270):285-9. View abstract
  87. Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease. Blood. 2016 Feb 18; 127(7):839-48. View abstract
  88. Dynamic Control of Enhancer Repertoires Drives Lineage and Stage-Specific Transcription during Hematopoiesis. Dev Cell. 2016 Jan 11; 36(1):9-23. View abstract
  89. Polycomb Repressive Complex 2 Is a Barrier to KRAS-Driven Inflammation and Epithelial-Mesenchymal Transition in Non-Small-Cell Lung Cancer. Cancer Cell. 2016 Jan 11; 29(1):17-31. View abstract
  90. Corrigendum: Failure to replicate the STAP cell phenomenon. Nature. 2016 Mar 17; 531(7594):400. View abstract
  91. Ezh2 regulates differentiation and function of natural killer cells through histone methyltransferase activity. Proc Natl Acad Sci U S A. 2015 Dec 29; 112(52):15988-93. View abstract
  92. LSD1 is essential for oocyte meiotic progression by regulating CDC25B expression in mice. Nat Commun. 2015 Dec 02; 6:10116. View abstract
  93. SWI/SNF-mutant cancers depend on catalytic and non-catalytic activity of EZH2. Nat Med. 2015 Dec; 21(12):1491-6. View abstract
  94. The LSD1 Family of Histone Demethylases and the Pumilio Posttranscriptional Repressor Function in a Complex Regulatory Feedback Loop. Mol Cell Biol. 2015 Dec; 35(24):4199-211. View abstract
  95. Failure to replicate the STAP cell phenomenon. Nature. 2015 Sep 24; 525(7570):E6-9. View abstract
  96. Hematopoietic stem cells develop in the absence of endothelial cadherin 5 expression. Blood. 2015 Dec 24; 126(26):2811-20. View abstract
  97. BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis. Nature. 2015 Nov 12; 527(7577):192-7. View abstract
  98. Hemoglobin switching's surprise: the versatile transcription factor BCL11A is a master repressor of fetal hemoglobin. Curr Opin Genet Dev. 2015 Aug; 33:62-70. View abstract
  99. Functional footprinting of regulatory DNA. Nat Methods. 2015 Oct; 12(10):927-30. View abstract
  100. EHMT1 and EHMT2 inhibition induces fetal hemoglobin expression. Blood. 2015 Oct 15; 126(16):1930-9. View abstract
  101. PRC2 Is Required to Maintain Expression of the Maternal Gtl2-Rian-Mirg Locus by Preventing De Novo DNA Methylation in Mouse Embryonic Stem Cells. Cell Rep. 2015 Sep 01; 12(9):1456-70. View abstract
  102. Inactivation of Eed impedes MLL-AF9-mediated leukemogenesis through Cdkn2a-dependent and Cdkn2a-independent mechanisms in a murine model. Exp Hematol. 2015 Nov; 43(11):930-935.e6. View abstract
  103. miRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction. Mol Ther. 2015 Sep; 23(9):1465-74. View abstract
  104. Regulation of Peripheral Nerve Myelin Maintenance by Gene Repression through Polycomb Repressive Complex 2. J Neurosci. 2015 Jun 03; 35(22):8640-52. View abstract
  105. Embryonic stem cells as sources of donor-independent platelets. J Clin Invest. 2015 Jun; 125(6):2261-3. View abstract
  106. BCL11A deletions result in fetal hemoglobin persistence and neurodevelopmental alterations. J Clin Invest. 2015 Jun; 125(6):2363-8. View abstract
  107. Opposing Roles for the lncRNA Haunt and Its Genomic Locus in Regulating HOXA Gene Activation during Embryonic Stem Cell Differentiation. Cell Stem Cell. 2015 May 07; 16(5):504-16. View abstract
  108. The mTORC1/4E-BP pathway coordinates hemoglobin production with L-leucine availability. Sci Signal. 2015 Apr 14; 8(372):ra34. View abstract
  109. Flow-induced protein kinase A-CREB pathway acts via BMP signaling to promote HSC emergence. J Exp Med. 2015 May 04; 212(5):633-48. View abstract
  110. 2014 William Allan Award: A hematologist's pursuit of hemoglobin genetics. Am J Hum Genet. 2015 Mar 05; 96(3):354-60. View abstract
  111. Angiopoietin-like proteins stimulate HSPC development through interaction with notch receptor signaling. Elife. 2015 Feb 25; 4. View abstract
  112. Functional Proteomic Analysis of Repressive Histone Methyltransferase Complexes Reveals ZNF518B as a G9A Regulator. Mol Cell Proteomics. 2015 Jun; 14(6):1435-46. View abstract
  113. Developmental control of polycomb subunit composition by GATA factors mediates a switch to non-canonical functions. Mol Cell. 2015 Jan 22; 57(2):304-316. View abstract
  114. Scl binds to primed enhancers in mesoderm to regulate hematopoietic and cardiac fate divergence. EMBO J. 2015 Mar 12; 34(6):759-77. View abstract
  115. Generation of genomic deletions in mammalian cell lines via CRISPR/Cas9. J Vis Exp. 2015 Jan 03; (95):e52118. View abstract
  116. Complementary genomic approaches highlight the PI3K/mTOR pathway as a common vulnerability in osteosarcoma. Proc Natl Acad Sci U S A. 2014 Dec 23; 111(51):E5564-73. View abstract
  117. Characterizing heterogeneity in leukemic cells using single-cell gene expression analysis. Genome Biol. 2014 Dec 03; 15(12):525. View abstract
  118. Mouse regulatory DNA landscapes reveal global principles of cis-regulatory evolution. Science. 2014 Nov 21; 346(6212):1007-12. View abstract
  119. A comparative encyclopedia of DNA elements in the mouse genome. Nature. 2014 Nov 20; 515(7527):355-64. View abstract
  120. Inflammatory signaling regulates embryonic hematopoietic stem and progenitor cell production. Genes Dev. 2014 Dec 01; 28(23):2597-612. View abstract
  121. Myeloproliferative neoplasms can be initiated from a single hematopoietic stem cell expressing JAK2-V617F. J Exp Med. 2014 Oct 20; 211(11):2213-30. View abstract
  122. TMEM14C is required for erythroid mitochondrial heme metabolism. J Clin Invest. 2014 Oct; 124(10):4294-304. View abstract
  123. Characterization of genomic deletion efficiency mediated by clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 nuclease system in mammalian cells. J Biol Chem. 2014 08 01; 289(31):21312-24. View abstract
  124. Reprogramming committed murine blood cells to induced hematopoietic stem cells with defined factors. Cell. 2014 Apr 24; 157(3):549-64. View abstract
  125. Distinct, strict requirements for Gfi-1b in adult bone marrow red cell and platelet generation. J Exp Med. 2014 May 05; 211(5):909-27. View abstract
  126. Corepressor Rcor1 is essential for murine erythropoiesis. Blood. 2014 May 15; 123(20):3175-84. View abstract
  127. Analysis of chromatin-state plasticity identifies cell-type-specific regulators of H3K27me3 patterns. Proc Natl Acad Sci U S A. 2014 Jan 21; 111(3):E344-53. View abstract
  128. Calpain 2 activation of P-TEFb drives megakaryocyte morphogenesis and is disrupted by leukemogenic GATA1 mutation. Dev Cell. 2013 Dec 23; 27(6):607-20. View abstract
  129. Distinct and combinatorial functions of Jmjd2b/Kdm4b and Jmjd2c/Kdm4c in mouse embryonic stem cell identity. Mol Cell. 2014 01 09; 53(1):32-48. View abstract
  130. GATA1s induces hyperproliferation of eosinophil precursors in Down syndrome transient leukemia. Leukemia. 2014 Jun; 28(6):1259-70. View abstract
  131. Polycomb repressive complex 2 regulates normal hematopoietic stem cell function in a developmental-stage-specific manner. Cell Stem Cell. 2014 Jan 02; 14(1):68-80. View abstract
  132. An erythroid enhancer of BCL11A subject to genetic variation determines fetal hemoglobin level. Science. 2013 Oct 11; 342(6155):253-7. View abstract
  133. Mapping cellular hierarchy by single-cell analysis of the cell surface repertoire. Cell Stem Cell. 2013 Oct 03; 13(4):492-505. View abstract
  134. Histone deacetylase inhibitors induce apoptosis in myeloid leukemia by suppressing autophagy. Leukemia. 2014 Mar; 28(3):577-88. View abstract
  135. Targeted disruption of the EZH2-EED complex inhibits EZH2-dependent cancer. Nat Chem Biol. 2013 Oct; 9(10):643-50. View abstract
  136. Delineating the mixed lineage leukemia gene expression network in hematopoietic stem cells. Proc Natl Acad Sci U S A. 2013 Jul 16; 110(29):11670-1. View abstract
  137. Histone demethylase Lsd1 represses hematopoietic stem and progenitor cell signatures during blood cell maturation. Elife. 2013 Jun 18; 2:e00633. View abstract
  138. Corepressor-dependent silencing of fetal hemoglobin expression by BCL11A. Proc Natl Acad Sci U S A. 2013 Apr 16; 110(16):6518-23. View abstract
  139. ETV1 directs androgen metabolism and confers aggressive prostate cancer in targeted mice and patients. Genes Dev. 2013 Mar 15; 27(6):683-98. View abstract
  140. Genome-wide association studies of hematologic phenotypes: a window into human hematopoiesis. Curr Opin Genet Dev. 2013 Jun; 23(3):339-44. View abstract
  141. Sustained PU.1 levels balance cell-cycle regulators to prevent exhaustion of adult hematopoietic stem cells. Mol Cell. 2013 Mar 07; 49(5):934-46. View abstract
  142. The switch from fetal to adult hemoglobin. Cold Spring Harb Perspect Med. 2013 Jan 01; 3(1):a011643. View abstract
  143. TiF1-gamma plays an essential role in murine hematopoiesis and regulates transcriptional elongation of erythroid genes. Dev Biol. 2013 Jan 15; 373(2):422-30. View abstract
  144. Altered hematopoiesis in trisomy 21 as revealed through in vitro differentiation of isogenic human pluripotent cells. Proc Natl Acad Sci U S A. 2012 Oct 23; 109(43):17567-72. View abstract
  145. Combinatorial assembly of developmental stage-specific enhancers controls gene expression programs during human erythropoiesis. Dev Cell. 2012 Oct 16; 23(4):796-811. View abstract
  146. Cyclin D3 coordinates the cell cycle during differentiation to regulate erythrocyte size and number. Genes Dev. 2012 Sep 15; 26(18):2075-87. View abstract
  147. Reawakening fetal hemoglobin: prospects for new therapies for the ß-globin disorders. Blood. 2012 Oct 11; 120(15):2945-53. View abstract
  148. Scl represses cardiomyogenesis in prospective hemogenic endothelium and endocardium. Cell. 2012 Aug 03; 150(3):590-605. View abstract
  149. Exome sequencing identifies GATA1 mutations resulting in Diamond-Blackfan anemia. J Clin Invest. 2012 Jul; 122(7):2439-43. View abstract
  150. The CACCC-binding protein KLF3/BKLF represses a subset of KLF1/EKLF target genes and is required for proper erythroid maturation in vivo. Mol Cell Biol. 2012 Aug; 32(16):3281-92. View abstract
  151. MAnorm: a robust model for quantitative comparison of ChIP-Seq data sets. Genome Biol. 2012 Mar 16; 13(3):R16. View abstract
  152. Polycomb repressive complex 2 is required for MLL-AF9 leukemia. Proc Natl Acad Sci U S A. 2012 Mar 27; 109(13):5028-33. View abstract
  153. MicroRNA-21 integrates pathogenic signaling to control pulmonary hypertension: results of a network bioinformatics approach. Circulation. 2012 Mar 27; 125(12):1520-32. View abstract
  154. MicroRNA-21 promotes fibrosis of the kidney by silencing metabolic pathways. Sci Transl Med. 2012 Feb 15; 4(121):121ra18. View abstract
  155. Haploinsufficiency of Dnmt1 impairs leukemia stem cell function through derepression of bivalent chromatin domains. Genes Dev. 2012 Feb 15; 26(4):344-9. View abstract
  156. A human stem cell model of early Alzheimer's disease pathology in Down syndrome. Sci Transl Med. 2012 Mar 07; 4(124):124ra29. View abstract
  157. Generation of a genomic reporter assay system for analysis of ?- and ß-globin gene regulation. FASEB J. 2012 Apr; 26(4):1736-44. View abstract
  158. PRC2 directly methylates GATA4 and represses its transcriptional activity. Genes Dev. 2012 Jan 01; 26(1):37-42. View abstract
  159. Dnmt3a silences hematopoietic stem cell self-renewal. Nat Genet. 2011 Dec 27; 44(1):13-4. View abstract
  160. Polycomb repressive complex 2 regulates normal development of the mouse heart. Circ Res. 2012 Feb 03; 110(3):406-15. View abstract
  161. Embryonic stem cell-specific signatures in cancer: insights into genomic regulatory networks and implications for medicine. Genome Med. 2011 Nov 29; 3(11):75. View abstract
  162. Zfp281 functions as a transcriptional repressor for pluripotency of mouse embryonic stem cells. Stem Cells. 2011 Nov; 29(11):1705-16. View abstract
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