Spring 2013 John H. Tietze Scientist Research Awards
The John H. Tietze Stem Cell Scientist Award is a one year award of $25,000 to support the research of any FACULTY member of ISCRM who is pursuing novel preliminary experiments, where the grant might provide sufficient stimulus to enable the research to advance to the point of being competitive for external funding. The research should involve or be relevant to some aspect of stem or progenitor cell biology or therapies.
Eirini Papapetrou, MD, PhD
Assistant Professor, Medicine/Hematology
University of Washington School of Medicine
FUNCTIONAL HUMAN GENETICS WITH DIRECTLY REPROGRAMMED AND GENETICALLY ENGINEERED HUMAN PLURIPOTENT STEM CELLS
My laboratory uses human pluripotent stem cells to study disorders of the blood and develop new therapeutics. Recently, a breakthrough technology, cell reprogramming, enabled us to generate pluripotent stem cells, called induced pluripotent stem cells (iPSCs), for the first time directly from adult cells. In a first wave of proof-of-principle studies we and others generated iPSCs from patients, carrying all the genetic information causing or predisposing to disease, and showed that we can differentiate them to specific cells and tissues where a disease manifests itself to study its mechanisms and to test drugs.
Our current focus is on myelodysplastic syndromes (MDS), blood disorders which, although common, remain poorly understood and treated and whose study is currently hampered by the lack of adequate biological tools (such as cell lines or animal models). We recently developed a new model of MDS using iPSCs derived from patient bone marrow and identified cellular behaviors (phenotypes) possibly specific to this disease. We plan to harness our model to investigate the pathogenesis of MDS and particularly to identify critical genes, which reside on a region of chromosome 7 that is frequently missing from patient bone marrow cells. This study can not only shed new light into this enigmatic disease but also provide a new paradigm of using iPSCs to discover new disease genes and study genetic mechanisms of human disease that are intractable in model organisms.