NEWS & EVENTS

Novel Molecular Etiologies of Dyserythropoiesis Workshop

6701 Rockledge Drive, (Conference Room 9100/9104)
Bethesda, MD 20892

Description

The severe anemia associated with hematologic disorders such as β thalassemia, myelodysplastic syndrome, and congenital dyserythropoietic anemia, arises from defects in late stage erythroid maturation (dyserythropoiesis). In contrast to anemias resulting from early stage defects, this group of disorders are resistant to erythropoietin (EPO) therapy since late-stage erythroblast differentiation and maturation is EPO independent. Transfusion, often associated with iron overload and alloimmunization, remains the principal approach to care. Fortunately, however, several molecular determinants of dyserythropoiesis have been recently identified. It is possible that these discoveries may reveal common pathways leading to dyserythropoiesis. This workshop will consider the gaps in our knowledge and technology that are barriers to our understanding of the underlying biologies that may lead to effective new treatments for late stage erythroid maturation defects. This workshop will also provide an opportunity for the research community to cooperatively consider the essential next steps for the field. 

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Agenda

8:30-8:40
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Welcome, Introductions and Charge to the Working Group

Drs. Donna DiMichele and Manjit Hanspal, NHBLI, NIH

8:40-8:50
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Introduction

Stefano Rivella, PhD, Children’s Hospital of Philadelphia

8:50-9:50
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SESSION I: Developmental switch from self-renewal to differentiation of erythroid cells

8:50-9:20, Orchestrating Developmental Signaling to Balance Erythroblast Proliferation and Differentiation

Emery Bresnick, PhD, University of Wisconsin-Madison
                           

9:20-9:50, Reconstructing Early Erythroid development in vivo using single-cell transcriptomics reveals a unique role for S phase

Merav Socolovsky, MD, PhD, University of Massachusetts Medical School

9:50-10:20
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SESSION II: Mechanisms affecting terminal erythropoiesis in disorders of β-thalassemia and MDS

Novel players in β-thalassemia dyserythropoiesis and new therapeutic strategies

Olivier Hermine, MD, PhD, Université Sorbonne Paris Cité

10:20-10:30
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Break

10:30-11:00
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Genetic investigation of the role of GDF11 in the treatment of β-thalassemia and MDS

Stefano Rivella, PhD, Children’s Hospital of Philadelphia

11:00-11:30
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Dyserythropoiesis due to haploinsufficiency of chromosome 7q gene in MDS

Amit Verma, MD, Albert Einstein College of Medicine

11:30-12:00
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Intrinsic and extrinsic factors in dyserythropoiesis in myelodysplastic syndromes

Peng Ji, MD, PhD, Northwestern University

12:00-12:30
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Innate immune pathway activation in Myelodysplastic Syndromes

Daniel T. Starczynowski, PhD, Cincinnati Children’s Hospital Medical Center

12:30-1:00
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Lunch

1:00-1:30
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FOXO3-Mediated Multilayer Regulation of Homeostatic and ß-thalassemic Erythropoiesis

Saghi Ghaffari, MD, PhD, Mount Sinai School of Medicine

1:30-2:30
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SESSION III: Iron overload and Ineffective erythropoiesis

1:30-2:00, Congenital sideroblastic anemias: iron and heme lost in mitochondrial translation

Mark D. Fleming, MD, Boston Children’s Hospital

2:00-2:30, Exogenous apo-transferrin and its effects on TfR1 in mouse models of β-thalassemia

Yelena Ginzburg, MD, Mount Sinai School of Medicine

2:30-2:45
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Break

2:45-4:30
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Prioritization of research topics for ineffective erythropoiesis; Identify gaps in knowledge and make recommendations on how to effectively address those gaps

Discussion leaders: Drs. Stefano Rivella and Saghi Ghaffari

4:30
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Adjourn