Cooperative Agreement U1AMB28550

This Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research federal grant program (CFDA 93.837), provides $306,870 to Kinetiq Therapeutics LLC, a life science startup in Texas, to conduct proof-of-concept studies on an enzyme replacement therapy (ERT) for Fanconi anemia. Fanconi anemia is a rare inherited disease characterized by bone marrow failure and malignancies. The project aims to demonstrate the feasibility of using an...

This Project Grant award of $390,000.00 from the National Institute of Diabetes and Digestive and Kidney Diseases (CFDA 93.847 Diabetes, Digestive, and Kidney Diseases Extramural Research) supports research on a novel mutation in the TASPASE1 gene and its impact on hematopoiesis and transcriptional regulation. The principal investigator at the University of Colorado-Denver is investigating how the TASPASE1V343M mutation disrupts proteolytic cleavage of the transcription factor TFIIA, leading...

This federal Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research federal grant program (CFDA 93.837), will provide $646,383 to Emory University to evaluate the impact of donor and recipient factors on the survival of transfused red blood cells (RBCs) in recipients with sickle cell disease. The research aims to examine mechanisms contributing to RBC clearance in both the recipient and the donor, including the impact of RBC...

This Project Grant award from the National Heart, Lung, and Blood Institute (CFDA 93.837 - Cardiovascular Diseases Research) provides $691,556 to The University of Texas Southwestern Medical Center (UT Southwestern) to determine the presence of amyloid in the blood and early cardiac amyloid infiltration in individuals of African ancestry who carry the V122I transthyretin (TTR) allele. The key objectives are to: 1) Assess the association between V122I TTR carrier status and circulating TTR...

This federal Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), part of the Cardiovascular Diseases Research program (CFDA 93.837), provides $300,000 to Gigamune, Inc. to develop an in vivo gene therapy for sickle cell disease. The project aims to test a novel lentiviral vector technology for efficiently editing hematopoietic stem cells to knockout the BCL11A enhancer, a key genetic target for sickle cell treatment. Through in vitro assays, the Phase I effort will...

This Project Grant award, valued at $801,000.00, was provided by the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) under the Diabetes, Digestive, and Kidney Diseases Extramural Research program (CFDA 93.847). The funding supports research at The Children's Hospital Corporation, doing business as Boston Children's Hospital, to investigate the role of stress granules in red blood cell development, particularly during stress conditions. The research aims to uncover the...

This federal Project Grant award of $225,101 from the National Cancer Institute under the Cancer Treatment Research program (CFDA 93.395) supports research to investigate the mechanisms underlying the increased risk of hematological malignancies in sickle cell disease patients. The key objectives of this 2-year project are to: 1) Perform experiments to understand how increased oxidative stress and DNA damage in hematopoietic stem and progenitor cells lead to the development of clonal...

This $445,000 Project Grant award from the National Heart Lung and Blood Institute (CFDA 93.837 Cardiovascular Diseases Research) supports research at The Children's Hospital of Philadelphia (CHOP) to investigate how the tropomyosin 1 gene product impacts the development of hematopoietic stem and progenitor cells and their precursors. The goal is to define genetic determinants of hematopoiesis that could enhance in vitro production of blood cells to support research and development of blood...

This Project Grant award from the National Heart, Lung, and Blood Institute (CFDA 93.837 - Cardiovascular Diseases Research) provides $765,622 to Yale University, with a sub-award to the Children's Hospital of Philadelphia, to develop orally bioavailable protein degraders targeting fetal hemoglobin gene repressors. The goal is to elevate fetal hemoglobin levels and prevent erythroid sickling to treat sickle cell disease. The award period runs from January 1, 2025 to November 30, 2028....

This $178,000 Project Grant awarded by the National Center for Advancing Translational Sciences (NCATS), a component of the National Institutes of Health (NIH), aims to develop a bone marrow organoid system to study RUNX1-familial platelet disorder with associated myeloid malignancy (RUNX1-FPD/MM). RUNX1-FPD/MM is a rare genetic disorder that can lead to thrombocytopenia, platelet dysfunction, and a high risk of developing myelodysplastic syndrome and leukemia. The project, awarded to The...