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Please join us as we continue our Bench to Bedside Series and discuss cutting edge research into therapies for sickle cell disease. Sickle cell disease is a complex, heterogenous genetic disorder that affects approximately 100,000 Americans and can result in both chronic organ dysfunction and acute life-threatening complications. We’ll be joined by David Williams, MD, whose work focuses on the biology of hematopoietic stem cells and selecting genetic and post-translational targets in sickle cell disease, as well as Martin Steinberg, MD, whose work has focused extensively on the pathophysiologic and epidemiologic features of this protean disease.

Department of Medicine Grand Rounds
June 8, 2021

Topic: BCL11A and Hb F in SCD/lentivirus vector containing a silencing RNA

“Targeting Fetal Hemoglobin in Sickle Cell Disease”
Martin Steinberg, MD
Professor of Medicine, Pediatrics, Pathology and Laboratory Medicine
Boston University School of Medicine

“Clinical Application of Post-transcriptional Silencing of BCL11A”
David Williams, MD
Chief of Hematology/Oncology
Boston Children’s Hospital
Leland Fikes Chair of Pediatrics
Harvard Medical School

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