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Active Grants and Sponsored Research

Sponsor Grant ID: DRG2021 Project Date: September 1, 2021 - August 31, 2024
Sponsoring Organization: The Edward P. Evans Foundation (EvansMDS)

Role of ANKRD26 Mutations in Familial MDS Predisposition

Description of Major Goals
The 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.
Sponsor Grant ID: 1R01HL159106-01 Project Date: August 1, 2021 - July 31, 2024
Sponsoring Organization: NIH/NHLBI

Bone Marrow Spatial Transcriptomics to Enhance In Vitro Platelet Production

Description of Major Goals
The 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.
Sponsor Grant ID: SG-0126-21-00 Project Date: July 1, 2021 - June 30, 2023
Sponsoring Organization: Harvard Stem Cell Institute

Bone Marrow Spatial Transcriptomics to Enhance In Vitro Platelet Production from Stem Cell Sources

Description of Major Goals
The 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.
Project Date: January 1, 2020 - December 31, 2022
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 Goals
The 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.
Sponsor Grant ID: TRP Project Date: July 1, 2021 - June 30, 2022
Sponsoring Organization: BOSTON CHILDREN'S HOSPITAL (TRP)

Use of CDK6 Inhbitors to Treat Familial Platelet Disorder with Propensity to Develop Leukemia

Description of Major Goals
The 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.
Project Date: January 1, 2021 - December 31, 2021
Sponsoring Organization: Elsa U Pardee]Foundation

Developing Novel Treatments for Juvenile Myelomonocytic Leukemia (JMML)

Description of Major Goals
The 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.
Sponsor Grant ID: 5R01DK098448-03 Project Date: September 15, 2013 - November 30, 2017
Sponsoring Organization: National Institutes Of Health (NIH)

Regulation of RUNX1 Multiprotein Complex Formation during Hematopoiesis

Description of Major Goals
The overall goal of this project is to further understand the mechanisms and signaling pathways that modulate RUNX1 protein-protein interactions during cellular differentiation.
Sponsor Grant ID: N/A Project Date: July 1, 2015 - September 30, 2017
Sponsoring Organization: Alex's Lemonade Stand Foundation

Transcriptional Effectors of Activated RAS Signaling in Juvenile Myelomonocytic Leukemia

Description of Major Goals
The 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.