IOX1 Fails to Reduce α-Globin and Mediates γ-Globin Silencing in Adult β0-Thalassemia/Hemoglobin E Erythroid Progenitor Cells
β-Thalassemia is an autosomal recessive blood disorder that presents with a quantitative abnormality of β-globin synthesis. Compound heterozygosity for β0-thalassemia and the β-globin chain variant (HBB: c.79G>A, hemoglobin E), hereinafter referred to as β0-thalassemia/HbE, has been characterized as one of the severe β-thalassemia genotypes caused by mutations within the β-globin gene [1]. This leads to the absence of β-globin chain and adult hemoglobin (HbA, α2β2) production, leaving unpaired α-globin chains in erythroid lineage cells. (Source: Experimental Hematology)
Source: Experimental Hematology - July 11, 2022 Category: Hematology Authors: Pinyaphat Khamphikham, Chokdee Wongborisuth, Sakorn Pornprasert, Adisak Tantiworawit, Amornrat Tangprasittipap, Duantida Songdej, Suradej Hongeng Tags: Brief Communication Source Type: research
IOX1 fails to reduce α-globin and mediates γ-globin silencing in adult β0-thalassemia/HbE erythroid progenitor cells
β-Thalassemia is an autosomal recessive blood disorder that presents with a quantitative abnormality of β-globin synthesis. Compound heterozygosity for β0-thalassemia and the β-globin chain variant (HBB: c.79G>A, hemoglobin E), hereinafter referred to as β0-thalassemia/HbE, has been characterized as one of the severe β-thalassemia genotypes caused by mutations within the β-globin gene (1). This leads to the absence of β-globin chain and adult hemoglobin (HbA, α2β2) production, leaving unpaired α-globin chains in erythroid lineage cells. (Source: Experimental Hematology)
Source: Experimental Hematology - July 11, 2022 Category: Hematology Authors: Pinyaphat Khamphikham, Chokdee Wongborisuth, Sakorn Pornprasert, Adisak Tantiworawit, Amornrat Tangprasittipap, Duantida Songdej, Suradej Hongeng Tags: Brief Communication Source Type: research
Germline ETV6 mutation promotes inflammation and disrupts lymphoid development of early hematopoietic progenitors
While the genomic landscape of acute lymphoblastic leukemia is being more clearly defined with large-scale, high-throughput sequencing studies,1,2 the molecular and cellular consequences of many of the identified aberrations are not fully understood. For example, ETV6 is among the most commonly altered genes in childhood B precursor acute lymphoblastic leukemia (B ALL), but our knowledge about the mechanisms by which ETV6 dysfunction promotes leukemogenesis is limited.3,4 This is of particular importance in light of the recent discovery of a rare autosomal dominant syndrome of thrombocytopenia and leukemia predisposition d...
Source: Experimental Hematology - July 6, 2022 Category: Hematology Authors: Chengjing Zhou, Rizvan Uluisik, Jesse W. Rowley, Camille David, Courtney L Jones, Christopher D Scharer, Leila Noetzli, Marlie H Fisher, Gregory D Kirkpatrick, Katrina Bark, Jeremy M Boss, Curtis J Henry, Eric M Pietras, Jorge Di Paola, Christopher C Port Source Type: research
FGD5 marks a subpopulation of hematopoietic stem and progenitor cells that resist interferon- γ-mediated differentiation
The hematopoietic system is composed of dozens of cell types starting from the most primitive hematopoietic stem cells (HSCs) to terminally differentiated lineage cells [1]. Even within canonically defined populations, one can find heterogeneity in gene expression and function [2,3]. Although all hematopoietic trees are defined to start at a single population of HSCs, there is an accumulating compendium of evidence that supports how diverse HSCs can be, in terms of both function and gene expression [4 –9]. (Source: Experimental Hematology)
Source: Experimental Hematology - June 25, 2022 Category: Hematology Authors: Daniel E. Morales-Mantilla, Katherine Y. King Tags: Article Source Type: research
FGD5 marks a subpopulation of HSPCs that resists IFN- γ-mediated differentiation
The hematopoietic system is comprised of dozens of cell types starting from the most primitive hematopoietic stem cells (HSCs) to terminally differentiated lineage cells 1. Even within canonically defined populations, one can find heterogeneity in gene expression and function 2,3. While all hematopoietic trees are defined to start at a single population of HSCs, there is an accumulating compendium of evidence that shows how diverse HSCs can be, both in terms of function and gene expression 4 –9. (Source: Experimental Hematology)
Source: Experimental Hematology - June 25, 2022 Category: Hematology Authors: Daniel E. Morales-Mantilla, Katherine Y. King Source Type: research
Cover 2: Editorial Board
(Source: Experimental Hematology)
Source: Experimental Hematology - June 15, 2022 Category: Hematology Source Type: research
RUNX1 Inhibition Using Lipid Nanoparticle-Mediated Silencing RNA Delivery as an Effective Treatment for Acute Leukemias
RUNX1 is one of three mammalian RUNX members, along with RUNX2 and RUNX3. RUNX1 contains a conserved Runt domain that mediates DNA binding and heterodimerization to their partner protein CBFB. RUNX1 and CBFB form a complex that regulates hematopoietic gene expression [1 –3]. Disruption of RUNX1 has been implicated in the development of hematopoietic neoplasms. Chromosomal abnormalities involving the RUNX1 gene are associated with several types of leukemia, including acute myeloid leukemia (AML) driven by RUNX1–RUNX1T1 [4,5]. (Source: Experimental Hematology)
Source: Experimental Hematology - May 25, 2022 Category: Hematology Authors: Kohei Iida, Akiho Tsuchiya, Moe Tamura, Keita Yamamoto, Shigehisa Kawata, Mitsuko Ishihara-Sugano, Motohiro Kato, Toshio Kitamura, Susumu Goyama Tags: Brief Communication Source Type: research
RUNX1 inhibition using lipid nanoparticle-mediated siRNA delivery as an effective treatment for acute leukemias
RUNX1 is one of three mammalian RUNX members, along with RUNX2 and RUNX3. RUNX1 contains a conserved Runt domain that mediates DNA binding and heterodimerization to their partner protein CBFB. RUNX1 and CBFB form a complex that regulates hematopoietic gene expression1-3. Disruption of RUNX1 has been implicated in the development of hematopoietic neoplasms. Chromosomal abnormalities involving the RUNX1 gene are associated with several types of leukemia, including acute myeloid leukemia (AML) driven by RUNX1-RUNX1T14,5. (Source: Experimental Hematology)
Source: Experimental Hematology - May 25, 2022 Category: Hematology Authors: Kohei Iida, Akiho Tsuchiya, Moe Tamura, Keita Yamamoto, Shigehisa Kawata, Mitsuko Ishihara-Sugano, Motohiro Kato, Toshio Kitamura, Susumu Goyama Tags: Category: Malignant Hematopoiesis Source Type: research
Cover 2: Editorial Board
(Source: Experimental Hematology)
Source: Experimental Hematology - May 19, 2022 Category: Hematology Source Type: research
Serum B-Cell Maturation Antigen is an Independent Prognostic Marker in Previously Untreated Chronic Lymphocytic Leukemia
Chronic lymphocytic leukemia (CLL) is a lymphoproliferative neoplasm of monoclonal B lymphocytes involving the peripheral blood, bone marrow and lymphoid organs.1 Of the 21,500 patients estimated to be diagnosed with CLL in 2022 in the United States, approximately 80% will have early-stage disease and will not need therapy at the time of diagnosis.2 The clinical course of CLL is highly heterogeneous, with some patients needing therapy soon after diagnosis while others remain in the “watch and wait” phase for many years. (Source: Experimental Hematology)
Source: Experimental Hematology - May 4, 2022 Category: Hematology Authors: Camilia M. Soof, Tanya M. Spektor, Sameer A. Parikh, Susan L. Slager, Kari G. Rabe, Timothy G. Call, Saad S. Kenderian, Wei Ding, Eli Muchtar, Matthew Ghermezi, Neil E. Kay, James R. Berenson Tags: Article Source Type: research
Cover 2: Editorial Board
(Source: Experimental Hematology)
Source: Experimental Hematology - April 29, 2022 Category: Hematology Source Type: research
m6A RNA modifications: key regulators of normal and malignant hematopoiesis
Hematopoiesis is tightly controlled at transcriptional and post-transcriptional levels. Alterations to these regulatory mechanisms directly affect the function of hematopoietic stem cells (HSCs), which are responsible for life-long blood cell production and maintenance, and this often results in the development of hematological malignancies, including acute myeloid leukemia (AML) [1]. (Source: Experimental Hematology)
Source: Experimental Hematology - April 26, 2022 Category: Hematology Authors: Pia Sommerkamp, Jessie A. Brown, Myriam L.R. Haltalli, Fran çois E. Mercier, Ly P. Vu, Kamil R. Kranc Tags: Perspective Source Type: research
Downregulation of SATB1 by miRNAs reduces megakaryocyte/erythroid progenitor expansion in preclinical models of Diamond –Blackfan anemia
Diamond –Blackfan Anemia (DBA) is a rare congenital bone marrow failure syndrome (estimated incidence of seven cases per million live births) with more than 90% of the reported cases presenting by 1 year of age. It is characterized by a macrocytic, moderate to severe anemia in association with hypoplastic bone marrow and reticulocytopenia. Almost half of DBA patients exhibit physical abnormalities, including short stature, craniofacial anomalies (50% of patients), upper limb and hand––in particular thumb (38%) abnormalities and genitourinary (39%) as well as cardiac (30%) abnormalities [1]. (Source: Experimental Hematology)
Source: Experimental Hematology - April 20, 2022 Category: Hematology Authors: Mark C. Wilkes, Vanessa Scanlon, Aya Shibuya, Alma-Martina Celika, Ascia Eskin, Zugen Chen, Anupama Narla, Bert Glader, Maria Grazia Roncarolo, Stanley F. Nelson, Kathleen M. Sakamoto Tags: Article Source Type: research
Downregulation of SATB1 by miRNAs Reduces Megakaryocyte/Erythroid Progenitor Expansion in pre-clinical models of Diamond Blackfan Anemia
Diamond Blackfan Anemia (DBA) is a rare congenital bone marrow failure syndrome (estimated incidence of seven cases per million live births) with more than 90% of the reported cases presenting by 1 year of age. It is characterized by a macrocytic, moderate to severe anemia in association with hypoplastic bone marrow and reticulocytopenia. Almost half of DBA patients exhibit physical abnormalities, including short stature, craniofacial anomalies (50% of patients), upper limb and hand ––in particular thumb (38%) abnormalities, and genitourinary (39%) as well as cardiac (30%) abnormalities (Da Costa et al., 2018). (Source...
Source: Experimental Hematology - April 20, 2022 Category: Hematology Authors: Mark C. Wilkes, Vanessa Scanlon, Aya Shibuya, Alma-Martina Celika, Ascia Eskin, Zugen Chen, Anupama Narla, Bert Glader, Maria Grazia Roncarolo, Stanley F. Nelson, Kathleen M. Sakamoto Tags: Article Source Type: research
Recurrent Transcriptional Responses in AML and MDS patients Treated with Decitabine
Summary: (Source: Experimental Hematology)
Source: Experimental Hematology - April 13, 2022 Category: Hematology Authors: Pawan Upadhyay, Jeremy Beales, Nakul M. Shah, Agata Gruszczynska, Christopher A. Miller, Allegra A. Petti, Sai Mukund Ramakrishnan, Daniel C. Link, Timothy J. Ley, John S. Welch Source Type: research
