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Journal Articles

BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

Barbara A. Konkle, Christopher E. Walsh, Miguel A. Escobar, Neil C. Josephson, Guy Young, Annette von Drygalski, Scott W. J. McPhee, R. Jude Samulski, Ivan Bilic, Maurus de la Rosa, Birgit M. Reipert, Hanspeter Rottensteiner, Friedrich Scheiflinger, John C. Chapin, Bruce Ewenstein, Paul E. Monahan

Blood (2021) 137 (6): 763–774.

https://doi.org/10.1182/blood.2019004625

Published: 2021

Includes: Supplemental data

Journal Articles

Sensing of Liver Iron Content Requires Cell-Cell Communication between Hepatocytes and Liver Sinusoidal Endothelial Cells

Silvia Colucci, Sandro Altamura, PhD, Matthias Hentze, Prof., Martina U. Muckenthaler, PhD

Blood (2019) 134 (Supplement_1): 432.

https://doi.org/10.1182/blood-2019-125586

Published: 2019

Journal Articles

NCOA4 Expression in Hepatic Cells Is Upregulated Under Physiological and Pathophysiological Conditions Associated with Hypoxia

Xiuqi Li, MS, Larisa Lozovatsky, MS, Karin E. Finberg, MD PhD

Blood (2019) 134 (Supplement_1): 431.

https://doi.org/10.1182/blood-2019-130858

Published: 2019

Journal Articles

FVIII Protein Is Not Detectable in Human PBMCs or Livers from Dogs with an Intron-22 Inversion Mutation: Implications for FVIII Immunogenicity and Tolerance

Devi Gunasekera, MBBS, Pooja Vir, PhD, Ahmad Faisal Karim, PhD, Margaret V. Ragni, MD MPH, Elizabeth P. Merricks, PhD, Timothy C. Nichols, MD, Kathleen P. Pratt, PhD

Blood (2019) 134 (Supplement_1): 630.

https://doi.org/10.1182/blood-2019-124880

Published: 2019

Journal Articles

The Immunophilin FKBP12 Inhibits Hepcidin By Modulating BMP Type I-Type II Receptors Interaction and Ligand Responsiveness

Alessandro Dulja, MSc, Alessia Pagani, PhD, Mariateresa Pettinato, Antonella Nai, PhD, Clara Camaschella, MD, Laura Silvestri, PhD

Blood (2019) 134 (Supplement_1): 430.

https://doi.org/10.1182/blood-2019-130058

Published: 2019

Journal Articles

Plasma EDA Fibronectin in Primary Myelofibrosis Correlates with Bone Marrow Fibrosis and Predicts Splenomegaly

Alessandro Malara, PhD, Cristian Gruppi, PhD, Margherita Massa, Vittorio Rosti, MD, Giovanni Barosi, Alessandra Balduini, MD

Blood (2019) 134 (Supplement_1): 1688.

https://doi.org/10.1182/blood-2019-130735

Published: 2019

Journal Articles

ATG5 Dependent Autophagy in Graft-Versus-Host Disease Target Organs Mitigates Disease Severity Post Allogeneic Bone Marrow Transplant

Austin Taylor, BA, Kay Oravecz-Wilson, Hideaki Fujiwara, Stephanie Kim, Dan Peltier, Molly Radosevich, Yaping Sun, Pavan Reddy

Blood (2019) 134 (Supplement_1): 1914.

https://doi.org/10.1182/blood-2019-131879

Published: 2019

Journal Articles

What Links Neutropenia to Immature Cardiolipin in Patients with Barth Syndrome (tafazzin-deficiency)?

Jihee Sohn, PhD, Thomas Brouse, Najihah Aziz, David B Sykes, MD PhD

Blood (2019) 134 (Supplement_1): 3579.

https://doi.org/10.1182/blood-2019-123680

Published: 2019

Journal Articles

Generation and Characterisation of KY1066, a Fully Human Antibody Targeting the Enzymatic Activity of Matriptase-2 for the Treatment of Iron Overload in Beta Thalassemia

Matthew Wake, PhD, Jonathan Papworth, BSc, Luke Bayliss, BSc, Benjamin Grimshaw, PhD, Natalie Rynkiewicz, PhD, Jemima Paterson, BSc, Alicia Poindron, MSc, Erin Spearing, Emily Carter, Robyne Hudson, BSc, Verena Petzer, MD, Amelie Largounez, BSc, Audrey Belot, BSc, Anais Palin, BSc, Igor Theurl, MD PhD, Volker Germaschewski, Delphine Meynard, PhD

Blood (2019) 134 (Supplement_1): 3532.

https://doi.org/10.1182/blood-2019-124075

Published: 2019

Journal Articles

Disbalanced Erythroid Ferroportin Expression Contributes to Ineffective Erythroid Output in Anemia of Chronic Disease

Verena Petzer, MD, Piotr Tymoszuk, PhD, Markus Seifert, Natascha Brigo, MSc, Philipp Grubwieser, Sylvia Berger, Richard Hilbe, Egon Demetz, PhD, Lara Valente de Souza, MD, Günter Weiss, Univ. Prof. Dr., Dominik Wolf, Univ. Prof. Dr., Igor Theurl, MD PhD

Blood (2019) 134 (Supplement_1): 3533.

https://doi.org/10.1182/blood-2019-131202

Published: 2019

Journal Articles

The Markedly Increased PAI1 in Obesity Induces a Compensatory Increase of Hepatocyte Tpa Expression By Activating a LRP1-CREB1 Pathway

Ze Zheng, MBBS, PhD, Keiko Nakamura, MD, Shana Gershbaum, Ira Tabas, MD PhD

Blood (2019) 134 (Supplement_1): 3625.

https://doi.org/10.1182/blood-2019-129271

Published: 2019

Journal Articles

Hemophilia A Dogs Tolerant to Human Factor VIII Provide a Unique Model to Determine Efficacy and Safety of AAV Delivery of Novel Factor VIII Variants

Giang N. Nguyen, BS, Lauren E. Wimsey, DVM, Elizabeth P. Merricks, PhD, Katherine P. Ponder, MD, Timothy C. Nichols, MD, Denise E. Sabatino, PhD

Blood (2019) 134 (Supplement_1): 3628.

https://doi.org/10.1182/blood-2019-126085

Published: 2019

Journal Articles

Evaluation of the Human Factor IX Gene Therapy Vector TAK-748 in Hemophilia: Results from Non-Clinical Studies in Factor IX Knockout Mice and Rhesus Monkeys

Markus Weiller, Helen Wang, Sogué Coulibaly, Maria Schuster, Hanspeter Rottensteiner, Kefeng Sun, Marinee K Chuah, Thierry Vandendriessche, Friedrich Scheiflinger, Werner Höllriegl

Blood (2019) 134 (Supplement_1): 4633.

https://doi.org/10.1182/blood-2019-124734

Published: 2019

Journal Articles

Design and Characterization of FLT210, a Potent Next Generation AAV-hFVIII Vector Candidate

Blood (2019) 134 (Supplement_1): 4638.

https://doi.org/10.1182/blood-2019-128490

Published: 2019

Journal Articles

Constitutively Activated STAT3 Induces the Expression of Gli1 in Chronic Lymphocytic Leukemia Cells

Uri Rozovski, MD, David M. Harris, PhD, Ping Li, PhD, Zhiming Liu, BS, Taghi Manshouri, PhD, Ivo Veletic, MD, Preetesh Jain, MBBS,MDDM,PhD, Alessandra Ferrajoli, MD, Jan A. Burger, MD PhD, Prithviraj Bose, MD, Philip A. Thompson, MBBS, Nitin Jain, MD, Srdan Verstovsek, MD PhD, William G. Wierda, MD PhD, Michael J Keating, MBBS, Zeev E. Estrov, MD

Blood (2019) 134 (Supplement_1): 3018.

https://doi.org/10.1182/blood-2019-124014

Published: 2019

Journal Articles

Hepatic Leukemia Factor Is Critical for Hematopoietic Stem Cells and Promotes MLL-AF9 Acute Myeloid Leukemia

Aurelie Baudet, PhD, Karolina Paulina Komorowska, PhD, Hultmark Simon, MS, Marcus Järås, PhD, Marion Chapellier, PhD, Gunnar Juliusson, MD PhD, Mattias Magnusson, PhD

Blood (2019) 134 (Supplement_1): 2466.

https://doi.org/10.1182/blood-2019-122661

Published: 2019

Journal Articles

Hyperphosphatemia Contributes to Inflammation and Iron Dysregulation in Models of Normal and Impaired Renal Function

Brian Czaya, BS, MSc, Beatrice Richter, PhD, Christopher Yanucil, Bsc, Isaac Campos, Bsc, Kylie Heitman, Bsc, Christian Faul, PhD

Blood (2019) 134 (Supplement_1): 2238.

https://doi.org/10.1182/blood-2019-122144

Published: 2019

Journal Articles

Biomarkers of Cardiopulmonary, Renal, and Liver Dysfunction in an Adult Sickle Cell Disease Cohort

Xu Zhang, PhD, Binal N. Shah, Roberto Machado, MD, Santosh L. Saraf, MD, Victor R Gordeuk, MD

Blood (2019) 134 (Supplement_1): 3574.

https://doi.org/10.1182/blood-2019-131813

Published: 2019

Journal Articles

Role of Exosomes in Hepcidin Regulation in β-Thalassemia

Marc Ruiz Martinez, PhD, Melanie Castro-Mollo, MD, Navneet Dogra, PhD, Wenbin An, MD, Ester Borroni, PhD, Antonia Follenzi, MD PhD, Thomas Coates, MD, Yelena Ginzburg, MD

Blood (2019) 134 (Supplement_1): 954.

https://doi.org/10.1182/blood-2019-131094

Published: 2019

Journal Articles

Targeting the BMP Receptor Inhibitor FKBP12 to Upregulate Hepcidin in Wild-Type and Hemojuvelin-Hemochromatosis Models

Mariateresa Pettinato, Mariam Aghajan, PhD, Violante Olivari, MSc, Alessandro Dulja, MSc, Antonella Nai, PhD, Jessica Bordini, PhD, Alessandro Campanella, PhD, Alessia Pagani, PhD, Shuling Guo, PhD, Clara Camaschella, MD, Laura Silvestri, PhD

Blood (2019) 134 (Supplement_1): 955.

https://doi.org/10.1182/blood-2019-126496

Published: 2019

NARROW

BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

Sensing of Liver Iron Content Requires Cell-Cell Communication between Hepatocytes and Liver Sinusoidal Endothelial Cells

NCOA4 Expression in Hepatic Cells Is Upregulated Under Physiological and Pathophysiological Conditions Associated with Hypoxia

FVIII Protein Is Not Detectable in Human PBMCs or Livers from Dogs with an Intron-22 Inversion Mutation: Implications for FVIII Immunogenicity and Tolerance

The Immunophilin FKBP12 Inhibits Hepcidin By Modulating BMP Type I-Type II Receptors Interaction and Ligand Responsiveness

Plasma EDA Fibronectin in Primary Myelofibrosis Correlates with Bone Marrow Fibrosis and Predicts Splenomegaly

ATG5 Dependent Autophagy in Graft-Versus-Host Disease Target Organs Mitigates Disease Severity Post Allogeneic Bone Marrow Transplant

What Links Neutropenia to Immature Cardiolipin in Patients with Barth Syndrome (tafazzin-deficiency)?

Generation and Characterisation of KY1066, a Fully Human Antibody Targeting the Enzymatic Activity of Matriptase-2 for the Treatment of Iron Overload in Beta Thalassemia

Disbalanced Erythroid Ferroportin Expression Contributes to Ineffective Erythroid Output in Anemia of Chronic Disease

The Markedly Increased PAI1 in Obesity Induces a Compensatory Increase of Hepatocyte Tpa Expression By Activating a LRP1-CREB1 Pathway

Hemophilia A Dogs Tolerant to Human Factor VIII Provide a Unique Model to Determine Efficacy and Safety of AAV Delivery of Novel Factor VIII Variants

Evaluation of the Human Factor IX Gene Therapy Vector TAK-748 in Hemophilia: Results from Non-Clinical Studies in Factor IX Knockout Mice and Rhesus Monkeys

Design and Characterization of FLT210, a Potent Next Generation AAV-hFVIII Vector Candidate

Constitutively Activated STAT3 Induces the Expression of Gli1 in Chronic Lymphocytic Leukemia Cells

Hepatic Leukemia Factor Is Critical for Hematopoietic Stem Cells and Promotes MLL-AF9 Acute Myeloid Leukemia

Hyperphosphatemia Contributes to Inflammation and Iron Dysregulation in Models of Normal and Impaired Renal Function

Biomarkers of Cardiopulmonary, Renal, and Liver Dysfunction in an Adult Sickle Cell Disease Cohort

Role of Exosomes in Hepcidin Regulation in β-Thalassemia

Targeting the BMP Receptor Inhibitor FKBP12 to Upregulate Hepcidin in Wild-Type and Hemojuvelin-Hemochromatosis Models

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American Society of Hematology

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ASH Publications

American Society of Hematology

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