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Benjamin Levine Ebert, MD, PhD


Medical Oncology / Hematologic Neoplasia

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Physician

  • George P. Canellos, MD, and Jean S. Canellos Professor of Medicine, Harvard Medical School
  • Chair of Medical Oncology, Dana-Farber Cancer Institute
  • Investigator, Howard Hughes Medical Institute
  • Institute Member, Broad Institute of Harvard and MIT

Clinical Interests

  • Hematology
  • Oncology

Contact Information

  • Office Phone Number(617) 632-1902

Bio

Dr. Benjamin Ebert is the George P. Canellos, MD, and Jean S. Canellos Professor of Medicine at Harvard Medical School, Chair of Medical Oncology at the Dana-Farber Cancer Institute, a Howard Hughes Medical Institute Investigator, and an Institute Member of the Broad Institute.Dr. Ebert is an elected member of the National Academy of Medicine, the American Society for Clinical Investigation and the Association of American Physicians. He served as President of the American Society for Clinical Investigation. His awards include the Till and McCollough Award from the International Society of Experimental Hematopoiesis, the William Dameshek Prize from the American Society of Hematology, the Meyenburg Prize, and mentoring and teaching awards from Harvard Medical School.Dr. Ebert received a bachelor's degree from Williams College and a doctorate from Oxford University as a Rhodes Scholar where he worked with Peter Ratcliffe, who was subsequently awarded the Nobel Prize in Medicine. He completed an M.D. from Harvard Medical School, a residency in internal medicine at Massachusetts General Hospital, and a fellowship in hematology/oncology at the Dana-Farber Cancer Institute. He was on the faculty of Brigham and Women’s Hospital for 10 years before returning to the Dana-Farber.

Board Certification:

  • Hematology, 2004
  • Internal Medicine, 2002
  • Medical Oncology, 2005

Fellowship:

  • Dana-Farber Cancer Institute, Hematology/Oncology
  • The Broad Institute of Harvard and MIT

Residency:

  • Massachusetts General Hospital, Internal Medicine

Medical School:

  • Harvard Medical School

Recent Awards:

  • Elected to American Society for Clinical Investigation
  • Elected to Association of American Physicians
  • Thomas A. McMahon Mentoring Award, Harvard-MIT Health Sciences and Technology Program
  • Elected to National Academy of Medicine
  • Meyenburg Prize for Cancer Research

Research

The primary focus of the Ebert laboratory is the genetics, biology, and therapy of myeloid malignancies.

The Ebert laboratory focuses on the molecular basis and treatment of hematologic malignancies and its non-malignant precursor conditions, with a particular focus on myelodysplastic syndromes (MDS) and clonal hematopoiesis of indeterminate potential (CHIP). The laboratory uses human genetics and a range of model systems to examine the genetics, biology, and clinical implications of MDS and CHIP. CHIP is a pre-malignant state that is associated with increased risk of hematologic malignancy, increased all-cause mortality, atherosclerotic cardiovascular disease, and changes in inflammatory markers. The lab studies the biological basis of transformation of hematopoietic cells by somatic mutations and has developed novel models to study myeloid malignancies.

The Ebert laboratory demonstrated that lenalidomide, a derivative of thalidomide, binds the CRL4-CRBN E3 ubiquitin ligase and induces degradation of specific substrates, a novel mechanism of action for a drug. More broadly, thalidomide analogs have the potential to induce degradation of a broad array of zinc finger transcription factors and other proteins. Expanding the library of molecules that function by degrading disease-relevant proteins, many of which were previously considered “undruggable,” has potential for novel therapeutics.

Association of Diet Quality With Prevalence of Clonal Hematopoiesis and Adverse Cardiovascular Events. JAMA Cardiol. 2021 Sep 01; 6(9):1069-1077.
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Systematic profiling of DNMT3A variants reveals protein instability mediated by the DCAF8 E3 ubiquitin ligase adaptor. Cancer Discov. 2021 Aug 24.
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Clonal hematopoiesis in patients receiving chimeric antigen receptor T-cell therapy. Blood Adv. 2021 08 10; 5(15):2982-2986.
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ZBTB33 is mutated in clonal hematopoiesis and myelodysplastic syndromes and impacts RNA splicing. Blood Cancer Discov. 2021 Sep; 2(5):500-517.
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Supplemental Association of Clonal Hematopoiesis With Incident Heart Failure. J Am Coll Cardiol. 2021 07 06; 78(1):42-52.
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Hematopoietic mosaic chromosomal alterations increase the risk for diverse types of infection. Nat Med. 2021 06; 27(6):1012-1024.
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Direct Exposure of Welders to Welding Fumes and Effect of Fume Extraction Systems Under Controlled Conditions. J Occup Environ Med. 2021 06 01; 63(6):490-502.
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Author Correction: Implications of TP53 allelic state for genome stability, clinical presentation and outcomes in myelodysplastic syndromes. Nat Med. 2021 May; 27(5):927.
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Leukemia vaccine overcomes limitations of checkpoint blockade by evoking clonal T cell responses in a murine acute myeloid leukemia model. Haematologica. 2021 05 01; 106(5):1330-1342.
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CBL mutations drive PI3K/AKT signaling via increased interaction with LYN and PIK3R1. Blood. 2021 04 22; 137(16):2209-2220.
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Whole exome sequencing of a breast tumor in a patient with Diamond Blackfan anemia. Blood Cells Mol Dis. 2021 07; 89:102566.
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Modeling and targeting of erythroleukemia by hematopoietic genome editing. Blood. 2021 03 25; 137(12):1628-1640.
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The AIM2 inflammasome exacerbates atherosclerosis in clonal haematopoiesis. Nature. 2021 04; 592(7853):296-301.
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Avadomide induces degradation of ZMYM2 fusion oncoproteins in hematologic malignancies. Blood Cancer Discov. 2021 May; 2(3):250-265.
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Cancer therapies based on targeted protein degradation - lessons learned with lenalidomide. Nat Rev Clin Oncol. 2021 07; 18(7):401-417.
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Author Correction: Inherited causes of clonal haematopoiesis in 97,691 whole genomes. Nature. 2021 Mar; 591(7851):E27.
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Author Correction: Implications of TP53 allelic state for genome stability, clinical presentation and outcomes in myelodysplastic syndromes. Nat Med. 2021 Mar; 27(3):562.
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Cohesin mutations alter DNA damage repair and chromatin structure and create therapeutic vulnerabilities in MDS/AML. JCI Insight. 2021 02 08; 6(3).
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A deep molecular response of splenic marginal zone lymphoma to front-line checkpoint blockade. Haematologica. 2021 02 01; 106(2):651-654.
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Reversible ON- and OFF-switch chimeric antigen receptors controlled by lenalidomide. Sci Transl Med. 2021 01 06; 13(575).
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Functional Genomics Identify Distinct and Overlapping Genes Mediating Resistance to Different Classes of Heterobifunctional Degraders of Oncoproteins. Cell Rep. 2021 01 05; 34(1):108532.
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Contribution of clonal hematopoiesis to adult-onset hemophagocytic lymphohistiocytosis. Blood. 2020 12 24; 136(26):3051-3055.
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TP53 mutations in myelodysplastic syndromes and secondary AML confer an immunosuppressive phenotype. Blood. 2020 12 10; 136(24):2812-2823.
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Small-molecule-induced polymerization triggers degradation of BCL6. Nature. 2020 12; 588(7836):164-168.
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Hematopoietic mosaic chromosomal alterations and risk for infection among 767,891 individuals without blood cancer. medRxiv. 2020 Nov 16.
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Premature Menopause, Clonal Hematopoiesis, and Coronary Artery Disease in Postmenopausal Women. Circulation. 2021 Feb 02; 143(5):410-423.
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Cancer therapy shapes the fitness landscape of clonal hematopoiesis. Nat Genet. 2020 11; 52(11):1219-1226.
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Calmodulin inhibitors improve erythropoiesis in Diamond-Blackfan anemia. Sci Transl Med. 2020 10 21; 12(566).
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Inherited causes of clonal haematopoiesis in 97,691 whole genomes. Nature. 2020 10; 586(7831):763-768.
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An induced pluripotent stem cell model of Fanconi anemia reveals mechanisms of p53-driven progenitor cell differentiation. Blood Adv. 2020 10 13; 4(19):4679-4692.
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Fitness Landscape of Clonal Hematopoiesis Under Selective Pressure of Immune Checkpoint Blockade. JCO Precis Oncol. 2020; 4.
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Implications of TP53 allelic state for genome stability, clinical presentation and outcomes in myelodysplastic syndromes. Nat Med. 2020 10; 26(10):1549-1556.
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The Clinical Challenge of Clonal Hematopoiesis, a Newly Recognized Cardiovascular Risk Factor. JAMA Cardiol. 2020 Aug 01; 5(8):958-961.
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SF3B1-mutant MDS as a distinct disease subtype: a proposal from the International Working Group for the Prognosis of MDS. Blood. 2020 07 09; 136(2):157-170.
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Clonal hematopoiesis is associated with adverse outcomes in multiple myeloma patients undergoing transplant. Nat Commun. 2020 06 12; 11(1):2996.
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The CDK inhibitor CR8 acts as a molecular glue degrader that depletes cyclin K. Nature. 2020 09; 585(7824):293-297.
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CXCR4 Signaling Has a CXCL12-Independent Essential Role in Murine MLL-AF9-Driven Acute Myeloid Leukemia. Cell Rep. 2020 05 26; 31(8):107684.
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The impact of the COVID-19 pandemic on cancer care. Nat Cancer. 2020 May 20; 1-3.
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Clonal hematopoiesis and measurable residual disease assessment in acute myeloid leukemia. Blood. 2020 05 14; 135(20):1729-1738.
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Clonal hematopoiesis in donors and long-term survivors of related allogeneic hematopoietic stem cell transplantation. Blood. 2020 04 30; 135(18):1548-1559.
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Identification of germline variants in adults with hemophagocytic lymphohistiocytosis. Blood Adv. 2020 03 10; 4(5):925-929.
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A new opening on aortic stenosis: predicting prognosis with clonal haematopoiesis. Eur Heart J. 2020 02 21; 41(8):940-942.
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A cryptic imatinib-sensitive G3BP1-PDGFRB rearrangement in a myeloid neoplasm with eosinophilia. Blood Adv. 2020 02 11; 4(3):445-448.
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Rapid and deep-scale ubiquitylation profiling for biology and translational research. Nat Commun. 2020 01 17; 11(1):359.
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Treatment-Induced Tumor Dormancy through YAP-Mediated Transcriptional Reprogramming of the Apoptotic Pathway. Cancer Cell. 2020 01 13; 37(1):104-122.e12.
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CHIPing Away at Breast Cancer. J Natl Cancer Inst. 2020 01 01; 112(1):10-11.
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Increased mitochondrial apoptotic priming with targeted therapy predicts clinical response to re-induction chemotherapy. Am J Hematol. 2020 03; 95(3):245-250.
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Clonal hematopoiesis as a model for premalignant changes during aging. Exp Hematol. 2020 03; 83:48-56.
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Non-del(5q) myelodysplastic syndromes-associated loci detected by SNP-array genome-wide association meta-analysis. Blood Adv. 2019 11 26; 3(22):3579-3589.
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Myelodysplastic syndromes (MDS) occurring in Agent Orange exposed individuals carry a mutational spectrum similar to that of de novo MDS. Leuk Lymphoma. 2020 03; 61(3):728-731.
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Clonal hematopoiesis in human aging and disease. Science. 2019 11 01; 366(6465).
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Functional characterization of BRCC3 mutations in acute myeloid leukemia with t(8;21)(q22;q22.1). Leukemia. 2020 02; 34(2):404-415.
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Genomic Biomarkers to Predict Resistance to Hypomethylating Agents in Patients With Myelodysplastic Syndromes Using Artificial Intelligence. JCO Precis Oncol. 2019; 3.
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A dominant-negative effect drives selection of TP53 missense mutations in myeloid malignancies. Science. 2019 08 09; 365(6453):599-604.
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Clonal Hematopoiesis: Crossroads of Aging, Cardiovascular Disease, and Cancer: JACC Review Topic of the Week. J Am Coll Cardiol. 2019 07 30; 74(4):567-577.
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Recurrent genetic HLA loss in AML relapsed after matched unrelated allogeneic hematopoietic cell transplantation. Blood Adv. 2019 07 23; 3(14):2199-2204.
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CHIP, CCUS, and Other Acronyms: Definition, Implications, and Impact on Practice. Am Soc Clin Oncol Educ Book. 2019 Jan; 39:400-410.
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Gene-centric functional dissection of human genetic variation uncovers regulators of hematopoiesis. Elife. 2019 05 09; 8.
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Patterns of substrate affinity, competition, and degradation kinetics underlie biological activity of thalidomide analogs. Blood. 2019 07 11; 134(2):160-170.
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Engineered Bcor mutations lead to acute leukemia of progenitor B-1 lymphocyte origin in a sensitized background. Blood. 2019 06 13; 133(24):2610-2614.
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Intergenerational epigenetic inheritance of cancer susceptibility in mammals. Elife. 2019 04 09; 8.
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Genomic subtyping and therapeutic targeting of acute erythroleukemia. Nat Genet. 2019 04; 51(4):694-704.
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Impaired human hematopoiesis due to a cryptic intronic GATA1 splicing mutation. J Exp Med. 2019 05 06; 216(5):1050-1060.
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Stabilization of the Max Homodimer with a Small Molecule Attenuates Myc-Driven Transcription. Cell Chem Biol. 2019 05 16; 26(5):711-723.e14.
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A Murine Model of Chronic Lymphocytic Leukemia Based on B Cell-Restricted Expression of Sf3b1 Mutation and Atm Deletion. Cancer Cell. 2019 02 11; 35(2):283-296.e5.
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Rps14, Csnk1a1 and miRNA145/miRNA146a deficiency cooperate in the clinical phenotype and activation of the innate immune system in the 5q- syndrome. Leukemia. 2019 07; 33(7):1759-1772.
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TP53 mutation status divides myelodysplastic syndromes with complex karyotypes into distinct prognostic subgroups. Leukemia. 2019 07; 33(7):1747-1758.
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CHIPping Away at the Pathogenesis of Heart Failure. JAMA Cardiol. 2019 01 01; 4(1):5-6.
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Clonal Hematopoiesis of Indeterminate Potential. J Clin Oncol. 2019 02 10; 37(5):419-422.
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Jak-ing Up the Plaque's Lipid Core…and Even More. Circ Res. 2018 11 09; 123(11):1180-1182.
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Inhibition of Casein Kinase 1 Alpha in Acute Myeloid Leukemia. N Engl J Med. 2018 11 08; 379(19):1873-1874.
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Defining the human C2H2 zinc finger degrome targeted by thalidomide analogs through CRBN. Science. 2018 11 02; 362(6414).
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Generations of physician-scientists. J Clin Invest. 2018 10 01; 128(10):4208-4212.
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CHIP (Clonal Hematopoiesis of Indeterminate Potential): Potent and Newly Recognized Contributor to Cardiovascular Risk. Circulation. 2018 08 14; 138(7):666-668.
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Synthetic Lethal and Convergent Biological Effects of Cancer-Associated Spliceosomal Gene Mutations. Cancer Cell. 2018 08 13; 34(2):225-241.e8.
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Gli1+ Mesenchymal Stromal Cells Are a Key Driver of Bone Marrow Fibrosis and an Important Cellular Therapeutic Target. Cell Stem Cell. 2018 Aug 02; 23(2):308-309.
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Thalidomide promotes degradation of SALL4, a transcription factor implicated in Duane Radial Ray syndrome. Elife. 2018 08 01; 7.
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CrbnI391V is sufficient to confer in vivo sensitivity to thalidomide and its derivatives in mice. Blood. 2018 10 04; 132(14):1535-1544.
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Genome-wide screen identifies cullin-RING ligase machinery required for lenalidomide-dependent CRL4CRBN activity. Blood. 2018 09 20; 132(12):1293-1303.
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Predicting progression to AML. Nat Med. 2018 07; 24(7):904-906.
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PPM1D-truncating mutations confer resistance to chemotherapy and sensitivity to PPM1D inhibition in hematopoietic cells. Blood. 2018 09 13; 132(11):1095-1105.
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CDK6 Antagonizes p53-Induced Responses during Tumorigenesis. Cancer Discov. 2018 07; 8(7):884-897.
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Clonal Hematopoiesis Confers Predisposition to Both Cardiovascular Disease and Cancer: A Newly Recognized Link Between Two Major Killers. Ann Intern Med. 2018 07 17; 169(2):116-117.
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Increased neutrophil extracellular trap formation promotes thrombosis in myeloproliferative neoplasms. Sci Transl Med. 2018 04 11; 10(436).
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Clonal Hematopoiesis after Induction Chemotherapy for Acute Myeloid Leukemia. N Engl J Med. 2018 03 29; 378(13):1244-1245.
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A decade of progress in myelodysplastic syndrome with chromosome 5q deletion. Leukemia. 2018 07; 32(7):1493-1499.
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Association of mutations with morphological dysplasia in de novo acute myeloid leukemia without 2016 WHO Classification-defined cytogenetic abnormalities. Haematologica. 2018 04; 103(4):626-633.
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A phase I study of lenalidomide plus chemotherapy with mitoxantrone, etoposide, and cytarabine for the reinduction of patients with acute myeloid leukemia. Am J Hematol. 2018 02; 93(2):254-261.
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Agonistic targeting of TLR1/TLR2 induces p38 MAPK-dependent apoptosis and NF?B-dependent differentiation of AML cells. Blood Adv. 2017 Oct 24; 1(23):2046-2057.
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SETD2 alterations impair DNA damage recognition and lead to resistance to chemotherapy in leukemia. Blood. 2017 12 14; 130(24):2631-2641.
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Clonal Hematopoiesis and Atherosclerosis. N Engl J Med. 2017 10 05; 377(14):1401-1402.
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Multiplex CRISPR/Cas9-Based Genome Editing in Human Hematopoietic Stem Cells Models Clonal Hematopoiesis and Myeloid Neoplasia. Cell Stem Cell. 2017 10 05; 21(4):547-555.e8.
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Leukaemia: Vitamin C regulates stem cells and cancer. Nature. 2017 09 28; 549(7673):462-464.
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Prevalent premalignancy. Blood. 2017 08 10; 130(6):695-696.
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Proposed minimal diagnostic criteria for myelodysplastic syndromes (MDS) and potential pre-MDS conditions. Oncotarget. 2017 Sep 26; 8(43):73483-73500.
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Utility of CRISPR/Cas9 systems in hematology research. Exp Hematol. 2017 10; 54:1-3.
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Clonal Hematopoiesis and Risk of Atherosclerotic Cardiovascular Disease. N Engl J Med. 2017 07 13; 377(2):111-121.
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Introduction to a review series on precision hematology. Blood. 2017 07 27; 130(4):408-409.
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NPM1 mutation but not RUNX1 mutation or multilineage dysplasia defines a prognostic subgroup within de novo acute myeloid leukemia lacking recurrent cytogenetic abnormalities in the revised 2016 WHO classification. Am J Hematol. 2017 Jul; 92(7):E123-E124.
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Gli1+ Mesenchymal Stromal Cells Are a Key Driver of Bone Marrow Fibrosis and an Important Cellular Therapeutic Target. Cell Stem Cell. 2017 06 01; 20(6):785-800.e8.
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Functional screen of MSI2 interactors identifies an essential role for SYNCRIP in myeloid leukemia stem cells. Nat Genet. 2017 Jun; 49(6):866-875.
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Deletion of ribosomal protein genes is a common vulnerability in human cancer, especially in concert with TP53 mutations. EMBO Mol Med. 2017 04; 9(4):498-507.
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Doubling Down on Mutant RAS Can MEK or Break Leukemia. Cell. 2017 02 23; 168(5):749-750.
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Clinical Implications of Genetic Mutations in Myelodysplastic Syndrome. J Clin Oncol. 2017 Mar 20; 35(9):968-974.
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Introduction to Genomics in Hematologic Malignancy. J Clin Oncol. 2017 03 20; 35(9):927-928.
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Prognostic Mutations in Myelodysplastic Syndrome after Stem-Cell Transplantation. N Engl J Med. 2017 02 09; 376(6):536-547.
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Drug discovery for Diamond-Blackfan anemia using reprogrammed hematopoietic progenitors. Sci Transl Med. 2017 02 08; 9(376).
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Copy-number and gene dependency analysis reveals partial copy loss of wild-type SF3B1 as a novel cancer vulnerability. Elife. 2017 02 08; 6.
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Clonal Hematopoiesis Associated With Adverse Outcomes After Autologous Stem-Cell Transplantation for Lymphoma. J Clin Oncol. 2017 May 10; 35(14):1598-1605.
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Diagnosis and management of AML in adults: 2017 ELN recommendations from an international expert panel. Blood. 2017 01 26; 129(4):424-447.
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The genetics of myelodysplastic syndrome: from clonal haematopoiesis to secondary leukaemia. Nat Rev Cancer. 2017 01; 17(1):5-19.
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Clonal hematopoiesis. Semin Hematol. 2017 01; 54(1):43-50.
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The EMT regulator ZEB2 is a novel dependency of human and murine acute myeloid leukemia. Blood. 2017 01 26; 129(4):497-508.
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Physiologic Expression of Sf3b1(K700E) Causes Impaired Erythropoiesis, Aberrant Splicing, and Sensitivity to Therapeutic Spliceosome Modulation. Cancer Cell. 2016 09 12; 30(3):404-417.
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Functionally identifiable apoptosis-insensitive subpopulations determine chemoresistance in acute myeloid leukemia. J Clin Invest. 2016 10 03; 126(10):3827-3836.
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The Public Repository of Xenografts Enables Discovery and Randomized Phase II-like Trials in Mice. Cancer Cell. 2016 07 11; 30(1):183.
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miR-126 Regulates Distinct Self-Renewal Outcomes in Normal and Malignant Hematopoietic Stem Cells. Cancer Cell. 2016 Apr 11; 29(4):602-606.
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The Public Repository of Xenografts Enables Discovery and Randomized Phase II-like Trials in Mice. Cancer Cell. 2016 04 11; 29(4):574-586.
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Core Circadian Clock Genes Regulate Leukemia Stem Cells in AML. Cell. 2016 Apr 07; 165(2):303-16.
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MLL-AF9- and HOXA9-mediated acute myeloid leukemia stem cell self-renewal requires JMJD1C. J Clin Invest. 2016 Mar 01; 126(3):997-1011.
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Rps14 haploinsufficiency causes a block in erythroid differentiation mediated by S100A8 and S100A9. Nat Med. 2016 Mar; 22(3):288-97.
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miR-126 Regulates Distinct Self-Renewal Outcomes in Normal and Malignant Hematopoietic Stem Cells. Cancer Cell. 2016 Feb 08; 29(2):214-28.
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An engineered multicomponent bone marrow niche for the recapitulation of hematopoiesis at ectopic transplantation sites. J Hematol Oncol. 2016 Jan 25; 9:4.
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Lenalidomide: deciphering mechanisms of action in myeloma, myelodysplastic syndrome and beyond. Curr Opin Cell Biol. 2015 Dec; 37:61-7.
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The novel mechanism of lenalidomide activity. Blood. 2015 Nov 19; 126(21):2366-9.
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DMSO Increases Mutation Scanning Detection Sensitivity of High-Resolution Melting in Clinical Samples. Clin Chem. 2015 Nov; 61(11):1354-62.
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Single-cell RNA-seq reveals changes in cell cycle and differentiation programs upon aging of hematopoietic stem cells. Genome Res. 2015 Dec; 25(12):1860-72.
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EHMT1 and EHMT2 inhibition induces fetal hemoglobin expression. Blood. 2015 Oct 15; 126(16):1930-9.
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Lenalidomide induces ubiquitination and degradation of CK1a in del(5q) MDS. Nature. 2015 Jul 09; 523(7559):183-188.
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Phase 1/2 trial of vorinostat in patients with sickle cell disease who have not benefitted from hydroxyurea. Blood. 2015 Jun 04; 125(23):3668-9.
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Inactivation of HDAC1 or HDAC2 induces gamma globin expression without altering cell cycle or proliferation. Am J Hematol. 2015 Jul; 90(7):624-8.
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Drosophila glucome screening identifies Ck1alpha as a regulator of mammalian glucose metabolism. Nat Commun. 2015 May 21; 6:7102.
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Charting the "Splice" Routes to MDS. Cancer Cell. 2015 May 11; 27(5):607-9.
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Clonal hematopoiesis of indeterminate potential and its distinction from myelodysplastic syndromes. Blood. 2015 Jul 02; 126(1):9-16.
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Clonal hematopoiesis and blood-cancer risk. N Engl J Med. 2015 03 12; 372(11):1071-2.
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Niche-Based Screening in Multiple Myeloma Identifies a Kinesin-5 Inhibitor with Improved Selectivity over Hematopoietic Progenitors. Cell Rep. 2015 Feb 10; 10(5):755-770.
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Acute myeloid leukemia ontogeny is defined by distinct somatic mutations. Blood. 2015 Feb 26; 125(9):1367-76.
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Mutations in G protein ß subunits promote transformation and kinase inhibitor resistance. Nat Med. 2015 Jan; 21(1):71-5.
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Age-related clonal hematopoiesis associated with adverse outcomes. N Engl J Med. 2014 Dec 25; 371(26):2488-98.
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Perivascular Gli1+ progenitors are key contributors to injury-induced organ fibrosis. Cell Stem Cell. 2015 Jan 08; 16(1):51-66.
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Distinct effects of concomitant Jak2V617F expression and Tet2 loss in mice promote disease progression in myeloproliferative neoplasms. Blood. 2015 Jan 08; 125(2):327-35.
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Role of casein kinase 1A1 in the biology and targeted therapy of del(5q) MDS. Cancer Cell. 2014 Oct 13; 26(4):509-20.
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TET2 mutations predict response to hypomethylating agents in myelodysplastic syndrome patients. Blood. 2014 Oct 23; 124(17):2705-12.
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Rational design of highly active sgRNAs for CRISPR-Cas9-mediated gene inactivation. Nat Biotechnol. 2014 Dec; 32(12):1262-7.
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L-Leucine improves the anaemia in models of Diamond Blackfan anaemia and the 5q- syndrome in a TP53-independent way. Br J Haematol. 2014 Nov; 167(4):524-528.
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Somatic mutations predict poor outcome in patients with myelodysplastic syndrome after hematopoietic stem-cell transplantation. J Clin Oncol. 2014 Sep 01; 32(25):2691-8.
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Lenalidomide induces degradation of IKZF1 and IKZF3. Oncoimmunology. 2014; 3(7):e941742.
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National trends in hospitalizations for sickle cell disease in the United States following the FDA approval of hydroxyurea, 1998-2008. Med Care. 2014 Jul; 52(7):612-8.
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Generation of mouse models of myeloid malignancy with combinatorial genetic lesions using CRISPR-Cas9 genome editing. Nat Biotechnol. 2014 Sep; 32(9):941-6.
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MDS is a stem cell disorder after all. Cancer Cell. 2014 Jun 16; 25(6):713-4.
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Loss of function of TET2 cooperates with constitutively active KIT in murine and human models of mastocytosis. PLoS One. 2014; 9(5):e96209.
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Integrated genomic analysis illustrates the central role of JAK-STAT pathway activation in myeloproliferative neoplasm pathogenesis. Blood. 2014 May 29; 123(22):e123-33.
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Mutations in epigenetic regulators including SETD2 are gained during relapse in paediatric acute lymphoblastic leukaemia. Nat Commun. 2014 Mar 24; 5:3469.
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Csnk1a1 inhibition has p53-dependent therapeutic efficacy in acute myeloid leukemia. J Exp Med. 2014 Apr 07; 211(4):605-12.
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Musashi-2 controls cell fate, lineage bias, and TGF-ß signaling in HSCs. J Exp Med. 2014 Jan 13; 211(1):71-87.
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Genome-scale CRISPR-Cas9 knockout screening in human cells. Science. 2014 Jan 03; 343(6166):84-87.
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Lenalidomide causes selective degradation of IKZF1 and IKZF3 in multiple myeloma cells. Science. 2014 Jan 17; 343(6168):301-5.
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Niche-based screening identifies small-molecule inhibitors of leukemia stem cells. Nat Chem Biol. 2013 Dec; 9(12):840-848.
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Deletion 5q MDS: molecular and therapeutic implications. Best Pract Res Clin Haematol. 2013 Dec; 26(4):365-75.
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The orthosteric GABAA receptor ligand Thio-4-PIOL displays distinctly different functional properties at synaptic and extrasynaptic receptors. Br J Pharmacol. 2013 Oct; 170(4):919-32.
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Sinister symbiosis: pathological hematopoietic-stromal interactions in CML. Cell Stem Cell. 2013 Sep 05; 13(3):257-8.
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The emerging importance of ribosomal dysfunction in the pathogenesis of hematologic disorders. Leuk Lymphoma. 2014 Mar; 55(3):491-500.
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The biology and clinical impact of genetic lesions in myeloid malignancies. Blood. 2013 Nov 28; 122(23):3741-8.
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Diminutive somatic deletions in the 5q region lead to a phenotype atypical of classical 5q- syndrome. Blood. 2013 Oct 03; 122(14):2487-90.
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Toll-like receptor alterations in myelodysplastic syndrome. Leukemia. 2013 Sep; 27(9):1832-40.
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In Vivo RNAi screening identifies a leukemia-specific dependence on integrin beta 3 signaling. Cancer Cell. 2013 Jul 08; 24(1):45-58.
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NRAS mutations with low allele burden have independent prognostic significance for patients with lower risk myelodysplastic syndromes. Leukemia. 2013 Oct; 27(10):2077-81.
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Initial experience with L-leucine therapy in myelodysplastic syndromes with associated chromosome 5q deletion. Blood. 2013 May 23; 121(21):4428.
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Global H3K4me3 genome mapping reveals alterations of innate immunity signaling and overexpression of JMJD3 in human myelodysplastic syndrome CD34+ cells. Leukemia. 2013 Nov; 27(11):2177-86.
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Macrophages support pathological erythropoiesis in polycythemia vera and ß-thalassemia. Nat Med. 2013 Apr; 19(4):437-45.
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Depletion of Jak2V617F myeloproliferative neoplasm-propagating stem cells by interferon-a in a murine model of polycythemia vera. Blood. 2013 May 02; 121(18):3692-702.
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(R)-2-hydroxyglutarate is sufficient to promote leukemogenesis and its effects are reversible. Science. 2013 Mar 29; 339(6127):1621-5.
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New strategies in myelodysplastic syndromes: application of molecular diagnostics to clinical practice. Clin Cancer Res. 2013 Apr 01; 19(7):1637-43.
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Implantable microenvironments to attract hematopoietic stem/cancer cells. Proc Natl Acad Sci U S A. 2012 Nov 27; 109(48):19638-43.
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Mitochondrial Atpif1 regulates haem synthesis in developing erythroblasts. Nature. 2012 Nov 22; 491(7425):608-12.
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Heterodimeric JAK-STAT activation as a mechanism of persistence to JAK2 inhibitor therapy. Nature. 2012 Sep 06; 489(7414):155-9.
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Molecular pathophysiology of myelodysplastic syndromes. Annu Rev Pathol. 2013 Jan 24; 8:21-47.
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Myeloproliferative neoplasm animal models. Hematol Oncol Clin North Am. 2012 Oct; 26(5):1065-81.
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miR-433 is aberrantly expressed in myeloproliferative neoplasms and suppresses hematopoietic cell growth and differentiation. Leukemia. 2013 Feb; 27(2):344-52.
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Validation of a prognostic model and the impact of mutations in patients with lower-risk myelodysplastic syndromes. J Clin Oncol. 2012 Sep 20; 30(27):3376-82.
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Gene sets identified with oncogene cooperativity analysis regulate in vivo growth and survival of leukemia stem cells. Cell Stem Cell. 2012 Sep 07; 11(3):359-72.
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L-Leucine improves the anemia and developmental defects associated with Diamond-Blackfan anemia and del(5q) MDS by activating the mTOR pathway. Blood. 2012 Sep 13; 120(11):2214-24.
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Novel approaches to the treatment of sickle cell disease: the potential of histone deacetylase inhibitors. Expert Rev Hematol. 2012 Jun; 5(3):303-11.
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Distinct roles for long-term hematopoietic stem cells and erythroid precursor cells in a murine model of Jak2V617F-mediated polycythemia vera. Blood. 2012 Jul 05; 120(1):166-72.
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Pulmonary fibrosis, bone marrow failure, and telomerase mutation. N Engl J Med. 2012 Apr 19; 366(16):1551-3.
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Stress granules contribute to a-globin homeostasis in differentiating erythroid cells. Biochem Biophys Res Commun. 2012 Apr 20; 420(4):768-74.
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Mutations in RNA splicing machinery in human cancers. N Engl J Med. 2011 Dec 29; 365(26):2534-5.
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Power cut: inhibiting mitochondrial translation to target leukemia. Cancer Cell. 2011 Nov 15; 20(5):555-6.
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Analysis of genomic aberrations and gene expression profiling identifies novel lesions and pathways in myeloproliferative neoplasms. Blood Cancer J. 2011 Nov; 1(11):e40.
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Translational medicine: ribosomopathies. Blood. 2011 Oct 20; 118(16):4300-1.
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Correction of sickle cell disease in adult mice by interference with fetal hemoglobin silencing. Science. 2011 Nov 18; 334(6058):993-6.
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Molecular dissection of the 5q deletion in myelodysplastic syndrome. Semin Oncol. 2011 Oct; 38(5):621-6.
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Stem cell gene expression programs influence clinical outcome in human leukemia. Nat Med. 2011 Aug 28; 17(9):1086-93.
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Coordinate loss of a microRNA and protein-coding gene cooperate in the pathogenesis of 5q- syndrome. Blood. 2011 Oct 27; 118(17):4666-73.
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Clinical effect of point mutations in myelodysplastic syndromes. N Engl J Med. 2011 Jun 30; 364(26):2496-506.
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Dexamethasone and lenalidomide have distinct functional effects on erythropoiesis. Blood. 2011 Aug 25; 118(8):2296-304.
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Diamond Blackfan anemia and ribosome biogenesis: introduction. Semin Hematol. 2011 Apr; 48(2):73-4.
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Ribosome defects in disorders of erythropoiesis. Int J Hematol. 2011 Feb; 93(2):144-149.
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Human tumors instigate granulin-expressing hematopoietic cells that promote malignancy by activating stromal fibroblasts in mice. J Clin Invest. 2011 Feb; 121(2):784-99.
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Densely interconnected transcriptional circuits control cell states in human hematopoiesis. Cell. 2011 Jan 21; 144(2):296-309.
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The STAT5 inhibitor pimozide decreases survival of chronic myelogenous leukemia cells resistant to kinase inhibitors. Blood. 2011 Mar 24; 117(12):3421-9.
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Unraveling the molecular pathophysiology of myelodysplastic syndromes. J Clin Oncol. 2011 Feb 10; 29(5):504-15.
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STATistical power of clonal analysis: differential STAT1 pathway activation downstream of the JAK2V617F mutation. Cancer Cell. 2010 Nov 16; 18(5):405-6.
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Haploinsufficiency for ribosomal protein genes causes selective activation of p53 in human erythroid progenitor cells. Blood. 2011 Mar 03; 117(9):2567-76.
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Genetic deletions in AML and MDS. Best Pract Res Clin Haematol. 2010 Dec; 23(4):457-61.
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Transcriptional analysis of HIV-specific CD8+ T cells shows that PD-1 inhibits T cell function by upregulating BATF. Nat Med. 2010 Oct; 16(10):1147-51.
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Musashi-2 regulates normal hematopoiesis and promotes aggressive myeloid leukemia. Nat Med. 2010 Aug; 16(8):903-8.
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NF1 inactivation revs up Ras in adult acute myelogenous leukemia. Clin Cancer Res. 2010 Aug 15; 16(16):4074-6.
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Chemical genetic strategy identifies histone deacetylase 1 (HDAC1) and HDAC2 as therapeutic targets in sickle cell disease. Proc Natl Acad Sci U S A. 2010 Jul 13; 107(28):12617-22.
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Physiological Jak2V617F expression causes a lethal myeloproliferative neoplasm with differential effects on hematopoietic stem and progenitor cells. Cancer Cell. 2010 Jun 15; 17(6):584-96.
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The genetic basis of myelodysplastic syndromes. Hematol Oncol Clin North Am. 2010 Apr; 24(2):295-315.
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Preface. The biology and treatment of myelodysplastic syndrome. Hematol Oncol Clin North Am. 2010 Apr; 24(2):xiii-xvi.
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The connectivity map links iron regulatory protein-1-mediated inhibition of hypoxia-inducible factor-2a translation to the anti-inflammatory 15-deoxy-delta12,14-prostaglandin J2. Cancer Res. 2010 Apr 15; 70(8):3071-9.
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Bone progenitor dysfunction induces myelodysplasia and secondary leukaemia. Nature. 2010 Apr 08; 464(7290):852-7.
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The Apc(min) mouse has altered hematopoietic stem cell function and provides a model for MPD/MDS. Blood. 2010 Apr 29; 115(17):3489-97.
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Ribosomopathies: human disorders of ribosome dysfunction. Blood. 2010 Apr 22; 115(16):3196-205.
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The landscape of somatic copy-number alteration across human cancers. Nature. 2010 Feb 18; 463(7283):899-905.
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Therapeutic potential of targeting haploinsufficient genes in cancer. Pediatr Blood Cancer. 2009 Aug; 53(2):131-2.
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Genetic characterization of TET1, TET2, and TET3 alterations in myeloid malignancies. Blood. 2009 Jul 02; 114(1):144-7.
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Deletion 5q in myelodysplastic syndrome: a paradigm for the study of hemizygous deletions in cancer. Leukemia. 2009 Jul; 23(7):1252-6.
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A germline JAK2 SNP is associated with predisposition to the development of JAK2(V617F)-positive myeloproliferative neoplasms. Nat Genet. 2009 Apr; 41(4):455-9.
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Ultrasome: efficient aberration caller for copy number studies of ultra-high resolution. Bioinformatics. 2009 Apr 15; 25(8):1078-9.
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Small-molecule inhibitors of HIF-2a translation link its 5'UTR iron-responsive element to oxygen sensing. Mol Cell. 2008 Dec 26; 32(6):838-48.
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Expression profiles of human 11beta-hydroxysteroid dehydrogenases type 1 and type 2 in inflammatory bowel diseases. Mol Cell Endocrinol. 2009 Mar 25; 301(1-2):104-8.
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Identification of an evolutionarily conserved transcriptional signature of CD8 memory differentiation that is shared by T and B cells. J Immunol. 2008 Aug 01; 181(3):1859-68.
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MicroRNA-mediated control of cell fate in megakaryocyte-erythrocyte progenitors. Dev Cell. 2008 Jun; 14(6):843-53.
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An erythroid differentiation signature predicts response to lenalidomide in myelodysplastic syndrome. PLoS Med. 2008 Feb; 5(2):e35.
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Identification of RPS14 as a 5q- syndrome gene by RNA interference screen. Nature. 2008 Jan 17; 451(7176):335-9.
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Identification of driver and passenger mutations of FLT3 by high-throughput DNA sequence analysis and functional assessment of candidate alleles. Cancer Cell. 2007 Dec; 12(6):501-13.
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pVHL acts as an adaptor to promote the inhibitory phosphorylation of the NF-kappaB agonist Card9 by CK2. Mol Cell. 2007 Oct 12; 28(1):15-27.
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Metagene projection for cross-platform, cross-species characterization of global transcriptional states. Proc Natl Acad Sci U S A. 2007 Apr 03; 104(14):5959-64.
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Failure to prolyl hydroxylate hypoxia-inducible factor alpha phenocopies VHL inactivation in vivo. EMBO J. 2006 Oct 04; 25(19):4650-62.
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Real-time elastography--an advanced method of ultrasound: First results in 108 patients with breast lesions. Ultrasound Obstet Gynecol. 2006 Sep; 28(3):335-40.
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MPLW515L is a novel somatic activating mutation in myelofibrosis with myeloid metaplasia. PLoS Med. 2006 Jul; 3(7):e270.
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Gene set enrichment analysis: a knowledge-based approach for interpreting genome-wide expression profiles. Proc Natl Acad Sci U S A. 2005 Oct 25; 102(43):15545-50.
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MicroRNA expression profiles classify human cancers. Nature. 2005 Jun 09; 435(7043):834-8.
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Activating mutation in the tyrosine kinase JAK2 in polycythemia vera, essential thrombocythemia, and myeloid metaplasia with myelofibrosis. Cancer Cell. 2005 Apr; 7(4):387-97.
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An RNA interference model of RPS19 deficiency in Diamond-Blackfan anemia recapitulates defective hematopoiesis and rescue by dexamethasone: identification of dexamethasone-responsive genes by microarray. Blood. 2005 Jun 15; 105(12):4620-6.
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Genomic approaches to hematologic malignancies. Blood. 2004 Aug 15; 104(4):923-32.
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