Volume 6.05 | Feb 10

Hematopoiesis News 6.05 February 10, 2015
Hematopoiesis News
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Chromothriptic Cure of WHIM Syndrome
Researchers report a case in which chromothripsis spontaneously cured a patient with WHIM syndrome, an autosomal dominant combined immunodeficiency disease caused by gain-of-function mutation of the chemokine receptor CXCR4. In this patient, deletion of the disease allele, CXCR4R334X, as well as 163 other genes from one copy of chromosome 2 occurred in a hematopoietic stem cell that repopulated the myeloid but not the lymphoid lineage. [Cell] Abstract | Graphical Abstract | Press Release
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PUBLICATIONS (Ranked by impact factor of the journal)
The Histone Demethylase Jarid1b Is Required for Hematopoietic Stem Cell Self-Renewal
Scientists found that Jarid1b is highly expressed in primitive hematopoietic compartments and is overexpressed in acute myeloid leukemias. Constitutive genetic deletion of Jarid1b did not impact steady-state hematopoiesis. [Blood] Abstract

Mutated Ptpn11 Alters Leukemic Stem Cell Frequency and Reduces the Sensitivity of Acute Myeloid Leukemia Cells to Mcl1 Inhibition
The authors report that Shp2 mutations occur in human acute myeloid leukemia (AML) at a rate of 6.6% in the ECOG E1900 dataset. They examined the role of mutated Shp2 in leukemias harboring myelomonocytic leukemia translocations which co-occur in human AML. [Leukemia] Abstract

NF-κB-Dependent DNA Damage-Signaling Differentially Regulates DNA Double-Strand Break Repair Mechanisms in Immature and Mature Human Hematopoietic Cells
Investigators explored the activity, quality, and molecular components of DNA double-strand-break repair in human hematopoietic stem and progenitor cell as compared with mature peripheral blood lymphocytes. [Leukemia] Abstract

Lentivirus Mediated Gene Transfer in Hematopoietic Stem Cells Is Impaired in SHIV-Infected, ART-Treated Nonhuman Primates
Scientists show that SHIV+, antiretroviral therapy (ART)-treated animals had very low gene marking levels after hematopoietic stem cell transplantation. [Mol Ther] Abstract

IGF-IR Determines the Fates of BCR/ABL Leukemia
Researchers investigated the role of insulin-like growth factor 1 receptor (IGF-IR) in chronic myeloid leukemia using the retroviral BCR/ABL transplantation mouse model. [J Hematol Oncol] Abstract | Full Article

Loss of Angiopoietin-Like 7 Diminishes the Regeneration Capacity of Hematopoietic Stem and Progenitor Cells
Investigators generated Angptl7-deficient mice by TALEN-mediated gene targeting and found that hematopoietic stem cell compartments in Angptl7-null mice were compromised. [J Hematol Oncol] Abstract | Full Article

Chronic Myeloid Leukemia-Derived Exosomes Promote Tumor Growth through an Autocrine Mechanism
In a chronic myeloid leukemia xenograft model, treatment of mice with exosomes caused a greater increase in tumor size compared with controls. [Cell Commun Signal] Abstract | Full Article


Allogeneic Hematopoietic Stem Cell Transplantation in Solid Organ Transplant Recipients: A Retrospective, Multicenter Study of the EBMT
Researchers conducted a questionnaire survey of the 565 European Society for Blood and Marrow Transplantation centers to analyze the outcome of allogeneic hematopoietic stem cell transplantation in recipients of solid organ transplantation. [Am J Transplant] Abstract

Extended Follow-Up of High-Dose Melphalan and Autologous SCT after Vincristine, Doxorubicin, Dexamethasone Induction in AL Amyloidosis of the Prospective Phase II HOVON-41 Study by the Dutch-Belgian Cooperative Trial Group for Hematology Oncology
Investigators evaluated the effect of three courses of vincristine, doxorubicin and dexamethasone followed by high-dose melphalan and autologous stem cell transplantation on an intention to treat basis. [Haematologica] Abstract | Full Article

Use of Eculizumab in Patients with Allogeneic Stem Cell Transplant-Associated Thrombotic Microangiopathy: A Study from the SFGM-TC
Thrombotic microangiopathy after allogeneic hematopoietic stem cell transplantation (HSCT) shares similarities with atypical hemolytic uremic syndrome (aHUS) in the underlying pathomechanisms. Eculizumab has been associated with impressive results in aHUS. Researchers retrospectively analyzed 12 patients who received eculizumab in France for severe post-HSCT thrombotic microangiopathy. [Transplantation] Abstract

Learn More: UM729, a Novel Small Molecule for Ex Vivo Expansion of Human HSCs
The Role of Oxygen in Hematopoietic Stem Cells Physiology
The authors cover the influence of physiologically relevant oxygen levels on adult stem cells through highlighting the research on the effect of oxygen concentration on hematopoietic stem cell maintenance, proliferation and differentiation. [J Cell Physiol] Abstract

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Cord Blood Registry and China Cord Blood Corporation Enter into Strategic Collaboration
Cord Blood Registry® and China Cord Blood Corporation China’s first and largest umbilical cord blood bank, entered into a memorandum of understanding regarding their strategic collaboration in China and the United States. [Cord Blood Registry®] Press Release

Sangamo BioSciences Announces FDA Acceptance of IND to Initiate Clinical Trial of its Novel ZFP Therapeutic® for Beta-Thalassemia
Sangamo BioSciences, Inc. announced that an Investigational New Drug (IND) application for the company’s SB-BCLmR-HSPC genome editing approach, which is designed to provide a one-time lasting therapy for beta-thalassemia, has been accepted by the U.S. Food and Drug Administration (FDA) and is now active. [Sangamo BioSciences, Inc.] Press Release

Three USC Researchers Win $4.3 Million in Awards from California’s Stem Cell Agency
Three scientists from Keck Medicine of USC have won grants exceeding $4.3 million from the California Institute for Regenerative Medicine for research that includes creating a temporary liver for patients, finding novel ways to treat immune disorders and blood diseases, and developing new animal models for heart failure, diabetes and neurodegenerative diseases. [University of Southern California] Press Release

Cord Blood Registry Announces Formation of Scientific & Medical Advisory Board
Cord Blood Registry® (CBR®) announced the establishment of a Scientific and Medical Advisory Board (SMAB). Composed of internationally renowned stem cell experts, the SMAB will provide input and guidance to help advance CBR’s mission to expand the scope of newborn stem cell therapies that may be available to patients and their families. [Cord Blood Registry®] Press Release

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European Medicines Agency (European Union)

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Therapeutic Goods Administration (Australia)
NEW EMBO | EMBL Symposium: Frontiers in Stem Cells & Cancer
March 29-31, 2015
Heidelberg, Germany

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NEW Senior Process Development Engineer (Sangamo BioSciences, Inc.)

NEW Director – Vector Production (Sangamo BioSciences, Inc.)

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.)

Scientist – Cell Culture Support Products (STEMCELL Technologies Inc.)

Research Associate – Cell Separation (STEMCELL Technologies Inc.)

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

Postdoctoral Fellow – Targeted Gene Editing in Hematopoietic Stem Cells (University Medical Center Freiburg)

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

Research Associate – Hematopoietic Stem Cells and Leukemia (City of Hope)

Postdoctoral Fellow – Hematopoiesis and Stem Cell Biology (Lund University)

Postdoctoral Research Scientist – Human T Cell Leukemia (Washington University School of Medicine)

Postdoctoral Research Fellow – Post-Transcriptional Gene Regulation Governing Cancer Cell Behavior (Fred Hutchinson Cancer Research Center)

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