Volume 7.09 | Mar 8

Hematopoiesis News 7.09 March 8, 2016
Hematopoiesis News
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Targeted Gene Addition in Human CD34+ Hematopoietic Cells for Correction of X-Linked Chronic Granulomatous Disease
Researchers demonstrated that temporally optimized delivery of zinc finger nuclease mRNA via electroporation and adeno-associated virus (AAV) 6 delivery of donor constructs in human hematopoietic stem and progenitor cells approaches clinically relevant levels of targeted integration into the AAVS1 safe harbor locus. [Nat Biotechnol] Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
PLZF Mutation Alters Mouse Hematopoietic Stem Cell Function and Cell Cycle Progression
Scientists found that Zbtb16lu/lu hematopoietic stem cells harbor a transcriptional signature associated with a loss of stemness and cell-cycle deregulation. [Blood] Abstract

Hematopoietic Reprogramming In Vitro Informs In Vivo Identification of Hemogenic Precursors to Definitive Hematopoietic Stem Cells
Researchers previously demonstrated that the induction of hematopoietic progenitors from fibroblasts progresses through hemogenic precursors that are Prom1+Sca1+CD34+CD45. Guided by these studies, they analyzed mouse placentas and identified a population with this phenotype. [Dev Cell]
Full Article | Graphical Abstract | Press Release

Rac1 GTPase Promotes Interaction of Hematopoietic Stem/Progenitor Cell with Niche and Participates in Leukemia Initiation and Maintenance in Mouse
By forced expression of constitutively active form of Rac1 in hematopoietic stem/progenitor cells (HSPCs), scientists demonstrated that active Rac1 promotes interaction of HSPC with niche. [Stem Cells] Abstract

Restriction of HIV-1-Based Lentiviral Vectors in Adult Primary Marrow-Derived and Peripheral Mobilized Human CD34+ Hematopoietic Stem and Progenitor Cells Occurs Prior to Viral DNA Integration
The authors demonstrated that the dominant point of restriction of an HIV-1-based lentiviral vector in adult human hematopoietic stem and progenitor cells from bone marrow and also those obtained following peripheral mobilization is prior to viral DNA integration. [Retrovirology] Full Article

Donor Dependent Variations in Hematopoietic Differentiation among Embryonic and Induced Pluripotent Stem Cell Lines
Researchers compared hematopoietic differentiation potentials from ES and iPS cell lines originated from various donors and derived them using integrative and non-integrative vectors. [PLoS One] Full Article


Haploidentical versus Matched-Sibling Transplant in Adults with Philadelphia-Negative High-Risk Acute Lymphoblastic Leukemia: A Biologically Phase III Randomized Study
Transplantation outcomes from haploidentical donors or matched-sibling donors were compared in the disease-specific, biologically Phase III randomized, multi-center study. [Clin Cancer Res] Abstract

Decreased Morbidity and Mortality of Autologous Hematopoietic Transplants for Children with Malignant Central Nervous System Tumors: The ‘Head Start’ Trials, 1991–2009
Two hundred and twenty-six children were enrolled on three consecutive ‘Head Start’ trials with primary malignant central nervous system tumors and underwent marrow-ablative chemotherapy. [Bone Marrow Transplant] Abstract

Outcome of Hematopoietic Stem Cell Transplantation Is Similar for Patients with a Partial In Vitro T-Cell-Depleted Graft compared with a Non-T-Cell-Depleted Graft when Stratified by the Refined Disease Risk Index
Researchers included 265 patients with partial T-cell-depleted graft (TDEP) and 163 non-TDEP patients in a retrospective study and compared outcomes following stratification using the refined disease risk index. [Bone Marrow Transplant] Abstract

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Notch Signaling: Its Roles and Therapeutic Potential in Hematological Malignancies
The authors give a general description of the Notch signaling pathway, and its physiologic role in hematopoiesis. They review the role of aberrant Notch signaling in several hematological malignancies. [Oncotarget] Full Article

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Top-Line Data from All Patients on the Primary Endpoint of Kiadis Pharma’s Phase II Trial with ATIR101™ Selected for Oral Presentation
Kiadis Pharma N.V. announced that key data from the company’s ongoing single-dose Phase II clinical trial with its lead product ATIR101™ has been selected for an oral presentation to discuss the top-line data. [Press release from Kiadis Pharma N.V. discussing research to be presented at the 42nd Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT), Valencia] Press Release

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Bio-Path Holdings Announces Completion of the Safety Segment of the Phase II Clinical Trial of BP1001 in Acute Myeloid Leukemia
Bio-Path Holdings, Inc. announced positive results from the eighth and final cohort of the safety segment of Bio-Path’s Phase II trial assessing the toxicity of the company’s lead product candidate, BP1001, in combination with low-dose cytarabine chemotherapy in patients with advanced acute myeloid leukemia. [Bio-Path Holdings, Inc.] Press Release

Baxalta Commences Phase I Clinical Trial of BAX 826, the Company’s Second Extended Half-Life Factor VIII Treatment for Hemophilia A
Baxalta Incorporated announced that it has dosed the first patient in its Phase I, first-in-human clinical trial of BAX 826, a recombinant Factor VIII treatment for hemophilia A that uses proprietary polysialic acid technology to extend its circulating half-life. [Baxalta Incorporated] Press Release

GlycoMimetics Announces Initial Clinical Data from Phase I Portion of Clinical Trial of GMI-1271 in AML Patients
GlycoMimetics, Inc. announced that of the first 13 evaluable patients in its clinical trial of GMI-1271 in combination with chemotherapy in patients with relapsed/refractory acute myeloid leukemia (AML), investigators have observed clinical responses in eight patients, for an overall response rate of 62%. [GlycoMimetics, Inc.] Press Release

True North Therapeutics Launches COMPASS Registry to Advance Development of New Therapeutics for Patients with Cold Agglutinin Disease (and Other Autoimmune Hemolytic Anemias)
True North Therapeutics announced the launch of the COMPASS Registry, a patient registry created by True North Therapeutics in collaboration with PatientCrossroads, for patients with Cold Agglutinin Disease (CAD). The COMPASS Registry is designed to build a deeper understanding of the natural history of CAD, characterize clinical biomarkers and engage early with patients and physicians to help accelerate development of new therapeutics. [True North Therapeutics] Press Release
National Institutes of Health (United States)

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European Medicines Agency (European Union)

Medicines and Healthcare Products Regulatory Agency (United Kingdom)

Therapeutic Goods Administration (Australia)
NEW International Society for Cellular Therapy (ISCT) 2016
May 25-28, 2016
Singapore, Singapore

Visit our events page to see a complete list of events in the hematopoiesis community.
NEW Associate Scientist – Hematopoietic Stem Cell Biology (Editas Medicine)

NEW Research Associate – Hematopoietic Stem Cell Biology (Editas Medicine)

NEW Postdoctoral Fellow Positions – Leukemogenesis (The University of Alabama)

Postdoctoral Fellow – Hematology/Oncology (Boston Children’s Hospital)

Postdoctoral Fellow – Cancer Stem Cell Biology (McGill University Health Center)

Postdoctoral Fellow – Cancer Biology (National University of Singapore)

PhD Position – Leukemogenesis and Treatment Resistance (Institute for Tumor Biology and Experimental Therapy)

Translational Research Scientist – Hematology/Oncology (University of Chicago)

Postdoctoral Associate – Epigenetic Regulation of Normal and Leukemic Stem Cells (Sanford Burnham Prebys Medical Discovery Institute)

Postdoctoral Researcher – Pre-Leukemic Stem Cells in the Thymus (Ghent University)

Assistant/Associate/Full Member Faculty – Myeloid Malignancies (Fred Hutchinson Cancer Research Center)

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