Transplantation across HLA barriers in aplastic anemia
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Goal 3: Advance Translational Research
Genome Editing and Gene Therapy
There is a critical need for the establishment of strategies that will determine the efficacy, safety, and toxicity of genome editing techniques specifically in hematologic diseases.
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Apheresis Medicine in the Management of Sickle Cell Disease
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How can we non-invasively, but still accurately, measure blood pressure in the pulmonary arteries?
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Would patients with pulmonary arterial hypertension (PAH) benefit from background anticoagulation in addition to their PAH-targe
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How can we increase the pharmaceutical clinical research of targeted therapies in pediatric PAH patients, including encouraging
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In pulmonary arterial hypertension (PAH), how can right ventricular function be improved in the setting of increased afterload
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Development of right ventricular-targeted therapies in pulmonary arterial hypertension (PAH)
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Anemia, oxygen delivery, and red blood cell transfusion
These questions are most relevant to critically ill populations that exhibit unique physiology, including those with low cardiac output (cardiac... more »
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Biology of Red Blood Cell Alloimmunization
This question encompasses: 1) the generation of alloantibodies that limit the availability of compatible blood or cause hemolytic disease of the fetus or newborn (HDFN); 2) the distinction between clinically significant and insignificant alloantibody responses, especially within alloantibody specificities... more »
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Evidence based approaches to Red Blood Cell transfusion
What are the optimal RBC transfusion thresholds for adult and pediatric cancer patients undergoing chemotherapy regimens that may improve functional status and quality of life?
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Epigenetics and Genomics
There is a need to investigate hemoglobin biosynthesis in order to develop novel approaches to treat sickle cell disease, thalassemia, and other anemias.
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Stem Cell Biology
There is a need to develop an artificial and functional hematopoietic stem cell (HSC) niche that allows for the expansion of repopulating HSCs.
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Goal 3: Advance Translational Research
Stem Cell Biology
There is a need to develop “designer platelets” and “designer red cells,” as well as facilitate large-scale production of these products for therapeutic and diagnostic use.