Active Grants and Sponsored Research
Sponsoring Organization: The Edward P. Evans Foundation (EvansMDS)
Role of ANKRD26 Mutations in Familial MDS Predisposition
Description of Major GoalsThe goal of this study is to further understand the mechanisms by which germline ANKRD26 mutations cause autosomal dominant thrombocytopenia and predisposition to MDS and leukemia through generation of mouse models and study of patient materials.
Sponsoring Organization: NIH/NHLBI
Bone Marrow Spatial Transcriptomics to Enhance In Vitro Platelet Production
Description of Major GoalsThe major goal of this project is to apply Multiplexed Error-Robust Fluorescent In
Situ Hybridization (MERFISH) spatial transcriptomic technology to further understand signaling events that occur when megakaryocytes interact with the bone marrow vascular sinusoidal niche and begin producing platelets. The long-term objective is to use this information to enhance in vitro platelet production from human induced pluripotent stem cells for transfusion medicine.
Situ Hybridization (MERFISH) spatial transcriptomic technology to further understand signaling events that occur when megakaryocytes interact with the bone marrow vascular sinusoidal niche and begin producing platelets. The long-term objective is to use this information to enhance in vitro platelet production from human induced pluripotent stem cells for transfusion medicine.
Sponsoring Organization: Harvard Stem Cell Institute
Bone Marrow Spatial Transcriptomics to Enhance In Vitro Platelet Production from Stem Cell Sources
Description of Major GoalsThe major goal of this project is to validate results from bone marrow Multiplexed
Error-Robust Fluorescent In Situ Hybridization (MERFISH) identified megakaryocyte pathways and their ability to enhance in vitro platelet production from stem cell sources.
Error-Robust Fluorescent In Situ Hybridization (MERFISH) identified megakaryocyte pathways and their ability to enhance in vitro platelet production from stem cell sources.
Sponsoring Organization: RUNX1 Research Program and Alex's Lemonade Stand Foundation
Pharmacologic Enhancement of Residual Wild Type RUNX1 Activity in Familial Platelet Disorder with Propensity to Develop Leukemia (FPD/AML)
Description of Major GoalsThe goal of this study is test the use of src family kinase inhibitors in preclinical models for use in enhancing the residual wild type RUNX1 activity in patients with heterozygous loss-of-function RUNX1 mutations.
Sponsoring Organization: BOSTON CHILDREN'S HOSPITAL (TRP)
Use of CDK6 Inhbitors to Treat Familial Platelet Disorder with Propensity to Develop Leukemia
Description of Major GoalsThe major goal of this project is to test several FDA-approved CDK4/6 inhibitors for their ability to rescue phenotypes associated with RUNX1 heterozygous loss of function mutations in models of RUNX1-related Familial Platelet Disorder with Propensity to Develop Leukemia.
Sponsoring Organization: Elsa U Pardee]Foundation
Developing Novel Treatments for Juvenile Myelomonocytic Leukemia (JMML)
Description of Major GoalsThe major goal of this study is to test the efficacy of a small molecule inhibitor of CBFb-RUNX1 interactions for its ability reduce disease severity in a mouse model of juvenile myelomonocytic leukemia (JMML) as well as in JMML patient sample cellular assays.
Sponsoring Organization: National Institutes Of Health (NIH)
Regulation of RUNX1 Multiprotein Complex Formation during Hematopoiesis
Description of Major GoalsThe overall goal of this project is to further understand the mechanisms and signaling pathways that modulate RUNX1 protein-protein interactions during cellular differentiation.
Sponsoring Organization: Alex's Lemonade Stand Foundation
Transcriptional Effectors of Activated RAS Signaling in Juvenile Myelomonocytic Leukemia
Description of Major GoalsThe goal of this project is to test the role of RUNX1 as a dysregulated transcriptional effect target of activated RAS/Shp2 signaling in Juvenile Myelomonocytic Leukemia.