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BUSM awarded $9 million to investigate treatment for sickle cell disease using iPS cells
Date:7/25/2011

(Boston) - Researchers from the Boston University School of Medicine (BUSM) were recently awarded a five-year $9 million grant from the National Heart, Lung, and Blood Institute (NHLBI) to mass-produce sickle cell anemia-specific induced Pluripotent Stem (iPS) cells. Under the direction of principal investigators Martin H. Steinberg, MD and George Murphy, PhD, the researchers propose making iPS cells from the blood of patients with sickle cell disease to better understand how certain genes are involved in the disease.

This initiative, which is being done in collaboration with Boston Medical Center (BMC), brings together two of the most dynamic entities at the Boston University Medical Campus: the Center of Excellence in Sickle Cell Disease, directed by Steinberg, and the Center for Regenerative Medicine (CReM), which is co-directed by Murphy, Gustavo Mostoslavsky, MD, PhD, and Darrell Kotton, MD.

iPS cells are derived by reprogramming adult cells into a primitive stem cell state. This process results in the creation of cells that are similar to embryonic stem (ES) cells in terms of their capability to differentiate into different types of cells. It is now widely accepted that iPS cells share many of the characteristics ES cells, including gene expression profiles, epigenetic signatures and pluripotency. iPS cells can be generated from mature somatic cells, such as skin or blood cells, allowing for the development of patient-specific cells and tissues that should not elicit inappropriate immune responses, making them a powerful tool for biological research and a resource for regenerative medicine.

Sickle cell anemia, an orphan disease of African Americans, is noted for its extensive morbidity and high mortality. Only one Food and Drug Administration (FDA) approved drug is available for its pathophysiologically based treatment. This agent, hydroxyurea, works through its ability to induce fetal hemoglobin (HbF) expression, which thwa
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Contact: Jenny Eriksen Leary
jenny.eriksen@bmc.org
617-638-6841
Boston University Medical Center
Source:Eurekalert

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