Completed Grants and Sponsored Research
Sponsoring Organization: American Heart Association
3D Chromatin Interaction Analysis to Accurately Map Cis-Regulatory Elements to Human Megakaryocyte/Platelet Genes
Description of Major GoalsGenerate maps that assign distal enhancer elements to their correct target gene(s) in human Mks
Sponsoring Organization: Boston Children's Hospital
Development of Mouse Model to Study RAS-mediated Elevation of Human Fetal Globin Levels
Description of Major GoalsThe goal of this project is to determine whether an inducible mouse model of activated RAS/PTPN11 signaling (PTPN11-lox-stopper-loxD61Y) containing a human beta-globin locus transgene recapitulates the elevated human fetal globin production seen in patients with disorders of activated RAS/PTPN11 signaling.
Sponsoring Organization: Harvard Stem Cell Institute
HSCI FACS Core Facility Award
Description of Major GoalsThis grant is to support a core facility that isolates normal and leukemia stem cells for investigators a Boston Children’s Hospital and the Harvard Stem Cell Institute.
Sponsoring Organization: National Institutes Of Health (NIH)
Megakaryocyte Transcription Factor Activation to Enhance In Vitro Platelet Production from Human IPSCs
Description of Major GoalsThe overall goal of this project is to investigate signaling pathways that activate megakaryocyte transcription factors as a means to enhance the efficiency of platelet production of human IPS cells.
Sponsoring Organization: Alex's Lemonade Stand Foundation and Babich Family Foundation
Pharmacologic Enhancement of Residual Wild Type RUNX1 Protein Activity in FPD/AML
Description of Major GoalsThe goal of this project is to identify small molecules that enhance residual wild type protein in individuals with germline heterozygous RUNX1 inactivating mutations in Familial Platelet Disorder with Propensity to Develop
Sponsoring Organization: National Insititutes of Health
Clinical Hematology Research Career Development Award
Description of Major GoalsThe major goal of this project is to support training in clinical research for non-malignant hematology. The long-term goal of this career development grant is to attract and retain talented young physician scientists to the field of blood diseases.
Sponsoring Organization: St. Baldrick'S Foundation
Role of GATA2 Dysregulation in Juvenile Myelmonocytic Leukemia
Description of Major GoalsJuvenile myelomonocytic leukemia is an aggressive blood cancer of young children. The only current curative treatment is bone marrow transplantation, Yet, about 50% of children still die from their disease despite this very aggressive therapy. An improved understanding of the mechanisms that cause JMML is critical to developing new treatments. Based on recent work, we hypothesize that a protein called GATA2, which normally turns on and off white blood cell genes, is erroneously activated in JMML. This proposal is designed to test this hypothesis. Positive results would support development of medicines to inhibit GATA2 for the treatment of JMML.
Sponsoring Organization: National Heart, Lung And Blood Institute (NHLBI)
Developmental Biology of Human Erythropoiesis: Project 4
Description of Major GoalsThe overall goal of this program project grant is to further understand the molecular regulation of erythroid development in humans. Dr. Cantor’s project involves elucidating the mechanisms by which GATA-1, a master erythroid transcription factor, distinguishes between direct target genes to activate versus repress and how it carries out these opposing transcriptional outputs in a gene context-dependent manner.
Sponsoring Organization: St. Baldrick'S Foundation
Role of RUNX1 (AML1) Dysregulation in Juvenile Myelomonocytic Leukemia
Description of Major GoalsThis proposal aims to further elucidate the molecular pathogenesis of an aggressive cancer of young children, with the goal of identifying improved and less-toxic treatments for this disorder
Sponsoring Organization: Harvard Stem Cell Institute
Runx1 Regulatory Mechanisms in Hematopoietic Stem Cell Ontogeny and Maintenance
Description of Major GoalsThe main goal of this study is to further elucidate the regulatory mechanisms that govern RUNX1’s activity in hematopoietic stem cell ontogeny and maintenance. It examines interactions between RUNX1 and Bmi1, and the role of src-family kinase mediated RUNX1 tyrosine phosphorylation.
Sponsoring Organization: Gabrielle's Angel Fund For Cancer Research
Inhibition of Runx1 Tyrosine Phosphorylation in the Treatment of Human Leukemia
Description of Major GoalsThe major goal of this study is to test whether clinically available src family tyrosine kinase inhibitors can block Runx1 tyrosine phosphorylation and enhance its activity.
Sponsoring Organization: Department Of Defense (U.S.) (DOD)
Runx-1 Centered Transcriptional Pathways as Tools to Discover Novel Genetic Risk Factors for Radiation-Induced Myelodysplastic Syndrome and Leukemia
Description of Major GoalsThe major goal of this study is to identify the genetic cause of families with autosomal dominant thrombocytopenia and propensity to develop leukemia who do not have Runx-1 gene alterations.
Sponsoring Organization: Boston Children's Hospital
Role ASXL1 Mutations in Human Myelodysplastic Syndrome and Leukemia
Description of Major GoalsThe aims of this study are to identify novel ASXL1 interacting proteins to further understand its function in hematopoiesis, and to test the hypothesis that mutant ASXL1 proteins associated with human leukemia have dominant negative activity.
Sponsoring Organization: National Institutes of Health
Proteomic Approach to Further Understanding the Role of Runx-1 in Myelodysplasia
Description of Major GoalsThe goals of this study were to purify and characterize Runx-1 containing multiprotein complexes in hematopoietic cells and examine the effects of myelodysplastic syndrome (MDS) associated Runx-1 mutations on observed protein-protein interactions.
Sponsoring Organization: National Institutes of Health
Proteomic Study of Megakaryocyte Transcriptional Control
Description of Major GoalsThis goal of this study is to isolate GATA-1 containing multiprotein complexes from megakaryocytes, identify the components, and assess their functional significance in megakaryopoiesis.
Sponsoring Organization: American Society of Hematology
Transcriptional Regulation of Megakaryopoiesis
Description of Major GoalsThe main goal of this study was to further understand the transcriptional regulation of megakaryopoiesis by identifying novel GATA-1 associated proteins and assessing their functional significance in megakaryopoiesis.
Sponsoring Organization: National Institutes of Health
Role of FOG-1 in Hematopoiesis
Description of Major GoalsThe major goal of this project is to define the mechanism of action of the transcriptional cofactor FOG-1 in hematopoiesis through a structure/function analysis.