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 federal Project Grant award from the National Heart, Lung, and Blood Institute (CFDA 93.837 Cardiovascular Diseases Research) provides $169,156 to The Washington University in St. Louis to support a 5-year research program focused on optimizing hematopoietic stem cell transplantation (HSCT) to treat non-malignant diseases, particularly sickle cell disease (SCD). The key objectives are to: 1) Evaluate novel stem cell mobilization regimens combining VLA-4 and CXCR4 inhibition to generate...
This federal Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research program (CFDA 93.837), provides $637,526 to Duke University to conduct research on the role of sickle red blood cells (RBCs) in activating neutrophils and promoting vaso-occlusion in sickle cell disease (SCD). The key objectives of the research are to: 1) delineate the characteristics of sickle RBCs that contribute to neutrophil activation, 2) examine the...
This Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837) provides $465,000 in funding to Bloodworks, a non-profit organization based in Seattle, WA. The award supports research into the mechanisms by which chronic inflammation disrupts hemostatic function in sickle cell disease (SCD). Specifically, the project aims to investigate how TNF-driven inflammation in SCD blocks autophagy and mitophagy in...
This federal Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research program (CFDA 93.837), provides $146,876 to the Coriell Institute for Medical Research to develop new methods for expanding functional hematopoietic stem cells (HSCs) from cryopreserved umbilical cord blood (UCB) samples. The project aims to optimize an approach using PPAR-α agonists, which have been shown to significantly expand long-term functional UCB HSCs...
This Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research program (CFDA 93.837), provides $814,209 to the Fred Hutchinson Cancer Center (doing business as Seattle Cancer Care Alliance) to develop CD90-targeted nanoparticles for in vivo hematopoietic stem cell gene therapy. The key objectives are to: 1) optimize nanoparticle formulations loaded with gene-editing agents and functionalize them for targeting, 2) evaluate the...
This federal Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837) provides $2,654,903 to The Children's Hospital Corporation (doing business as Boston Children's Hospital) to develop a novel alpha-retroviral vector platform for in vivo and ex vivo delivery of gene therapy payloads to hematopoietic stem cells (HSCs). The key objectives are to: (1) develop integrating vectors to deliver a curative...
This Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837) provides $1,123,357.00 to support research on the bone marrow microenvironment and its role in regulating blood cell production. The research, to be conducted by the Children's Hospital Medical Center in Cincinnati, Ohio, aims to define the spatial organization and specialized vascular microenvironments within the bone marrow that recruit and...
This federal Project Grant award from the National Heart, Lung, and Blood Institute (CFDA 93.837 - Cardiovascular Diseases Research) provides $608,126 to the Regents of the University of Minnesota to develop and validate single-cell biomarkers for predicting clinical outcomes in sickle cell disease (SCD). The research aims to: 1) define the relationship between SCD adverse outcomes and single-cell biochemical/mechanical properties to identify the most promising biomarkers; 2) define the...
The National Heart, Lung, and Blood Institute (NHLBI) awarded a $802,301 Project Grant under CFDA 93.837 - Cardiovascular Diseases Research to Weill Medical College of Cornell University. The grant's objective is to define the mechanisms that control the stem cell niche, with the aim of inhibiting the expansion of mutated blood stem cells that can lead to increased risk of life-shortening conditions like strokes, heart disease, and blood cancers. The research will identify the signals that...