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National Heart, Lung, and Blood Institute

Application Receipt Date(s): May 08, 2012

The purpose of this U54 program issued by the National Heart, Lung, and Blood Institute (NHLBI) is to encourage interdisciplinary approaches to translational research in the hemoglobinopathies, providing opportunities for potentially high-impact research, which will translate basic observations to applied clinical research. Applicants will be required to propose research that will, by the end of the project period, develop candidate potentially therapeutic molecules, biomarkers, or imaging modalities. Applicants are expected to assemble interactive, multidisciplinary teams that have the combined expertise to formulate a plan for successful translation. Research teams are not required to have prior collaborative experience but must be able to demonstrate an integrated, practical approach that will result in the effective progression of mechanistic/basic concepts toward application in the clinic.

The proposed project should focus on the development of a specific discovery or deliverable (e.g., small molecules, biologics, biomarkers, and other methodologies) to be used in the diagnosis or treatment of sickle cell disease or the thalassemias.

The hemoglobinopathies, sickle cell disease and the thalassemias, are the most common clinically significant human monogenetic diseases globally. Although the causative mutations primarily affect the qualitative or quantitative production of hemoglobin, the resultant pathophysiologies result in dysregulation of inflammatory and thromboregulatory pathways leading to damage of numerous organ systems, a process beginning shortly after birth.

Research in the hemoglobinopathies has traditionally focused on the red cell and the hemoglobin molecule. During the past decade, investigators have contributed new insights through work in genomics and epigenetics, drug development, the neurobiology of pain, iron metabolism, vascular biology, patient-reported outcomes (PROs), and comparative effectiveness research (CER). However, there have been few opportunities for broad-based programs that would benefit from collaboration among hematologists and investigators in relevant areas. Attracting …

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