Journal
STEM CELL REPORTS
Volume 17, Issue 7, Pages 1509-1535Publisher
CELL PRESS
DOI: 10.1016/j.stemcr.2022.06.004
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Funding
- KAKENHI
- National Medical Research Council grant of Singapore Translational Research Investigator Award
- Dietmar Hopp Foundation
- National Institutes of Health [P01HL053749, R01HL156647, U19HL129903, R01HL148852, R01DK098263, R01HL069438, R01DK115577]
- Paul S. Frenette Scholar Awards Program of the Ruth L. and David S. Gottesman Institute for StemCell Research and Regenerative Medicine
- Leukemia & Lymphoma Society [1360-19]
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This article reviews the two research topics, sickle cell disease and the hematopoietic stem cell niche, that formed the basis of Dr. Paul S. Frenette's outstanding career. The first part discusses important discoveries in sickle cell disease pathophysiology and recent advances in its treatment. The second part focuses on the complex microenvironment within the bone marrow that controls hematopoietic stem cell function and homeostasis, highlighting key aspects of Dr. Frenette's research and his significant scientific contributions in both fields.
In this retrospective, we review the two research topics that formed the basis of the outstanding career of Dr. Paul S. Frenette. In the first part, we focus on sickle cell disease (SCD). The defining feature of SCD is polymerization of the deoxygenated mutant hemoglobin, which leads to a vicious cycle of hemolysis and vaso-occlusion. We survey important discoveries in SCD pathophysiology that have led to recent advances in treatment of SCD. The second part focuses on the hematopoietic stem cell (HSC) niche, the complex microenvironment within the bone marrow that controls HSC function and homeostasis. We detail the cells that constitute highlight key aspects of his research, and identify his most important scientific contributions in both fields.
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