Volume 6.09 | Mar 10

Hematopoiesis News 6.09 March 10, 2015
Hematopoiesis News
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Stem Cell Researchers Develop Promising Method to Treat Sickle Cell Disease
Stem cell researchers have shown that a novel stem cell gene therapy method could lead to a one-time, lasting treatment for sickle cell disease. The study outlines a method that corrects the mutated gene that causes sickle cell disease and shows, for the first time, that the gene correction method leads to the production of normal red blood cells. [Press release from UCLA discussing online publication in Blood]
Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Osteoblast Ablation Reduces Normal Long-Term Hematopoietic Stem Cell Self-Renewal but Accelerates Leukemia Development
Investigators used an osteoblast (OB) visualization and ablation mouse model to study the role of OBs in regulating normal hematopoietic stem cell and chronic myelogenous leukemic stem cells. [Blood] Abstract

Runx1 Is Required for Hematopoietic Defects and Leukemogenesis in Cbfb-MYH11 Knockin Mice
The authors showed that a modified CBFβ-SMMHC with decreased RUNX1 binding activity accelerates leukemogenesis. These results raise questions about the importance of RUNX1 in leukemogenesis by CBFβ-SMMHC. [Leukemia] Abstract

Expression of Hedgehog Pathway Mediator GLI Represents a Negative Prognostic Marker in Human Acute Myeloid Leukemia and Its Inhibition Exerts Anti-Leukemic Effects
Researchers investigated expression and prognostic impact of Hedgehog pathway members in acute myeloid leukemia (AML). Pre-treatment samples from 104 newly diagnosed AML patients were analyzed by qPCR and expression of Hedgehog family members was correlated to clinical outcome. [Clin Cancer Res] Abstract

Conserved and Divergent Aspects of Human T Cell Development and Migration in Humanized Mice
Investigators found that both conventional T cell development and intra-thymic migration patterns in humanized mice closely resemble human thymopoiesis. [Immunol Cell Biol] Abstract | Full Article

Role of Transgene Regulation in Ex Vivo Lentiviral Correction of Artemis Deficiency
Researchers compared the human Artemis promoter to the moderate-strength human PGK promoter and the strong human EF1α promoter to regulate expression of Artemis after ex vivo lentiviral transduction of hematopoietic stem cells in a murine model of severe combined immunodeficiency. [Hum Gene Ther] Abstract

Enhanced Hematopoietic Stem Cell Function Mediates Immune Regeneration following Sex Steroid Blockade
Investigators showed that, mechanistically, sex steroid ablation induces hematopoietic and lymphoid recovery by functionally enhancing both hematopoietic stem cell self-renewal and propensity for lymphoid differentiation through intrinsic molecular changes. [Stem Cell Reports] Full Article | Graphical Abstract


Haploidentical BMT and Post-Transplant Cy for Severe Aplastic Anemia: A Multicenter Retrospective Study
The use of post-transplant cyclophosphamide (Cy) has been shown to be an effective strategy to prevent GvHD in recipients of haploidentical hematopoietic SCT, but the majority of reports have focused on patients with hematology malignancies. Researchers describe the outcome of 16 patients who underwent haploidentical transplantation using a reduced-intensity conditioning regimen with post-transplant Cy. [Bone Marrow Transplant] Abstract

Co-Infusion of Mesenchymal Stromal Cells Has No Effect on Relapse and Infection in Leukemia Patients Undergoing Haploidentical Hematopoietic Stem Cell Transplantation
Investigators observed the outcomes of mesenchymal stromal cells co-infusion during haploidentical hematopoietic stem cell transplantation (haplo-HSCT) and compared the relapse and infection rates with patients receiving traditional haplo-HSCT. [Leuk Lymphoma] Abstract

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Development of Hematopoietic Stem and Progenitor Cells from Human Pluripotent Stem Cells
The authors discuss the current status in the generation of multipotent hematopoietic stem and progenitor cells from human pluripotent stem cells via hemogenic endothelial cells. They also review the achievements in direct reprogramming from non-hematopoietic cells to hematopoietic stem and progenitor cells. [J Cell Biochem] Abstract

Visit our reviews page to see a complete list of reviews in the hematopoiesis research field.
Spectrum Pharmaceuticals Announces FDA’s Acceptance of NDA Filing for Captisol-Enabled™ (Propylene Glycol-Free) Melphalan
Spectrum Pharmaceuticals announced that its New Drug Application (NDA) for captisol-enabled melphalan, has been accepted by the U.S. Food and Drug Administration (FDA). Spectrum is seeking FDA approval for its use as a high-dose conditioning treatment prior to autologous hematopoietic (progenitor) stem cell transplantation in patients with multiple myeloma, an orphan drug designation. [Spectrum Pharmaceuticals, Inc.] Press Release

AOP Orphan Pharmaceuticals Announces Progress of Pivotal Phase III Trial PROUD-PV Applying Ropeginterferon Alfa 2b, a Novel, Long-Acting, Mono-Pegylated Interferon for Treatment of Polycythemia Vera
AOP Orphan Pharmaceuticals AG reported the completion of recruitment for its Phase III trial PROUD-PV to support global licensure of ropeginterferon alfa 2b, a novel, long-acting, mono-pegylated Interferon for the treatment of Polycythemia Vera. [AOP Orphan Pharmaceuticals AG] Press Release

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National Institutes of Health (United States)

Food and Drug Administration (United States)

Center for Biologics Evaluation and Research (United States)

European Medicines Agency (European Union)

Medicines and Healthcare Products Regulatory Agency (United Kingdom)

Therapeutic Goods Administration (Australia)
NEW SelectBio Stem Cells in Drug Discovery
June 2-3, 2015
Cambridge, United Kingdom

Visit our events page to see a complete list of events in the hematopoiesis community.
NEW Research Scientist – Cell Signaling in Normal and Leukemic Hematopoietic Stem and Progenitor Cells (Indiana University)

Research Technologist – Hematopoietic Product Development (STEMCELL Technologies Inc.)

Principal Scientist – Hematopoietic Products (STEMCELL Technologies Inc.)

Scientist – Pluripotent Stem Cell Media Development, High Throughput Screening (STEMCELL Technologies Inc.)

Research Associate – Cell Separation (STEMCELL Technologies Inc.)

Postdoctoral Fellow – Stem Cell/Translational Medicine (Rutgers New Jersey Medical School)

Postdoctoral Position – Drug Development for Anemia (Lund University)

Postdoctoral Position – Pediatric Hematology and Oncology (University Medical Center of Freiburg)

Senior Process Development Engineer (Sangamo BioSciences, Inc.)

Director – Vector Production (Sangamo BioSciences, Inc.)

Postdoctoral Position – Hematopoietic Stem Cell Physiology and Pathophysiology (Kumamoto University)

Postdoctoral Position – Genetic Engineering and Transplantation of Hematopoietic Stem Cells (University of California – San Francisco)

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