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个人简介
I am a physician-scientist focused on improving therapeutic approaches for sickle cell disease and beta-thalassemia via study of key signaling pathways that regulate expression of the fetal form of hemoglobin. My graduate work at the University of Rochester School of Medicine focused on the injury and recovery of the red blood cell lineage in a sublethal radiation mouse model (Peslak et al. Exp. Hematol. 2011, Peslak et al. Blood 2012) and was supported by a Ruth L. Kirschstein National Research Service Award for Individual Predoctoral MD/PhD Fellows (F30, NIDDK). After completing my Internal Medicine residency at the Hospital of the University of Pennsylvania as part of the Physician-Scientist Program, I began my Hematology/Oncology fellowship at UPenn and continued my long-standing interest in red cell disorders with a focus on sickle cell disease (SCD) and thalassemia. I joined the lab of Dr. Gerd Blobel in September 2017 to conduct translational research in SCD, specifically studying the role of a recently identified regulator of fetal hemoglobin, heme-regulated inhibitor (HRI). Since joining the lab, I have found that HRI depletion combined with other pharmacologic inducers of fetal hemoglobin (HbF) leads to clinically significant expression of HbF that significantly reduces sickling in vitro. For this work, I have published a first-author manuscript in Blood Advances (Peslak et al. Blood Adv. 2020) as well as a manuscript in Blood detailing the underlying mechanism of HRI-mediated HbF regulation (Huang, Peslak et al. Blood 2020). Over the past two years, I have also received the UPenn Austrian Award for Basic Research by a Fellow and was awarded the Doris Duke Charitable Foundation Physician Scientist Fellowship, the UPenn Measey Foundation Physician Scientist Fellowship, and most recently an NIH Mentored Clinical Scientist Research Career Development (K08) Award and an American Society of Hematology Scholar Award.
My long-term goal is to improve therapeutic approaches for SCD and beta-thalassemia via mechanistic studies of key signaling pathways that regulate the expression of HbF. Toward this goal, I carried out a CRISPR-Cas9 based screen to identify additional potentially druggable molecules which might function alone or cooperatively with HRI in governing HbF production. I thus discovered the protein phosphatase PP6C as a novel HbF regulator. Of note, PP6C modulates the protein kinase GCN2, a close relative of HRI with similar substrates. My ongoing research to further explore the role of PP6C-mediated signaling pathways in HbF regulation is the focus of my current work. I am currently a faculty Instructor in Hematology at the University of Pennsylvania with a clinical focus in non-malignant hematology, particularly SCD and thalassemia. I plan to pursue a career as an adult red cell physician-scientist with an independent NIH-funded research program.
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Bloodno. 19 (2024): 1980-1991
BLOOD (2023): 2382
bioRxiv the preprint server for biology (2023)
NATURE GENETICS (2023)
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MOLECULAR THERAPY-NUCLEIC ACIDS (2023): 452-465
BLOODno. Supplement 1 (2023): 13-13
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