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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

Knockdown of HNF4A Gene Increases Fetal Hemoglobin Synthesis in Hudep-2

Ingrid Grazielle Sousa, BSc, MSc, Priscila Keiko Matsumoto Martin, PhD, Dulcinéia Martins de Albuquerque, BSc, PhD, Carolina Lanaro, BSc, PhD, Ryo Kurita, PhD, Yukio Nakamura, MD PhD, Fernando Ferreira Costa, MD PhD

Blood (2019) 134 (Supplement_1): 968.

https://doi.org/10.1182/blood-2019-130920

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

Journal Articles

Targeting Transferrin Receptor 2: A Novel "Erythropoiesis-Stimulating Approach" in a Model of Anemia of Chronic Kidney Disease

Violante Olivari, MSc, Maria Rosa Lidonnici, PhD, Mariateresa Pettinato, Laura Silvestri, PhD, Giuliana Ferrari, PhD, Clara Camaschella, MD, Antonella Nai, PhD

Blood (2019) 134 (Supplement_1): 956.

https://doi.org/10.1182/blood-2019-125783

Published: 2019

Journal Articles

High-Dose Dexamethasone (HD-DXM) Plus Oseltamivir Versus High-Dose Dexamethasone for Treatment of Adult Primary Immune Thrombocytopenia: Interim Analysis of a Prospective, Multicenter, Randomized, Controlled Trial

Lu Sun, PhD, Xinguang Liu, Hai Zhou, Hongguo Zhao, Chenglu Yuan, Daqi Li, Zhencheng Wang, Yu Hou, Jun Peng, Ming Hou

Blood (2019) 134 (Supplement_1): 900.

https://doi.org/10.1182/blood-2019-121825

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

Platelet and Immune Characteristics of Patients with Immune Thrombocytopaenia Non Responders to Therapeutic Treatments

Elena Monzón Manzano, Raul Justo Sanz, Diana Hernández, Teresa Álvarez Roman, Ihosvany Fernandez-Bello, Mónica Martín, Larissa Valor, María Isabel Rivas Pollmar, Miguel A Canales, MD PhD, Victor Jimenez-Yuste, Nora Butta, PhD

Blood (2019) 134 (Supplement_1): 1089.

https://doi.org/10.1182/blood-2019-125772

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

The Translocation t(7;12)(q36;p13) Induces Myeloid Leukemia in Immuno-Compromised but Not Immunocompetent Mice

Ahmed Waraky, PhD MSc, BSc, Anders Östlund, MSc, BSc, Laleh Arabanian, PhD, Tina Nilsson, MSc, BSc, Linda Fogelstrand, Lars Palmqvist, MD PhD

Blood (2019) 134 (Supplement_1): 2707.

https://doi.org/10.1182/blood-2019-123019

Published: 2019

Journal Articles

Recreation of t(7;12)(q36;p13) in Human Induced Pluripotent Stem Cells Using CRISPR/Cas9 Generates a Useful Model for Studying Acute Myeloid Leukemia

Tina Nilsson, MSc, BSc, Ahmed Waraky, PhD MSc, BSc, Anders Östlund, MSc, BSc, Laleh Arabanian, PhD, Julia Asp, PhD, Linda Fogelstrand, Lars Palmqvist, MD PhD

Blood (2019) 134 (Supplement_1): 2736.

https://doi.org/10.1182/blood-2019-123004

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

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

Suicide Gene Therapy with a CD33 Targeted AAV6 Vector Expressing an Inducible Caspase-9 Suicide Gene Is Therapeutic in a Xenotransplantation Model of Acute Myeloid Leukemia

Nusrat Khan, MSc, Sridhar Bammidi, Jayandharan Giridhara Rao, PhD

Blood (2019) 134 (Supplement_1): 3351.

https://doi.org/10.1182/blood-2019-125222

Published: 2019

Journal Articles

The Factor VIII Variant X5 Enhances Hemophilia a Gene Therapy Efficiency By Its Improved Secretion

Franziska Horling, PhD, Johannes Lengler, PhD, Wenjing Cao, PhD, Biao Dong, PhD, Bagirath Gangadharan, Maurus De La Rosa, PhD, Werner Hoellriegl, PhD, Birgit M Reipert, PhD, Friedrich Scheiflinger, Weidong Xiao, PhD, Hanspeter Rottensteiner

Blood (2019) 134 (Supplement_1): 3356.

https://doi.org/10.1182/blood-2019-126489

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

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

Knockdown of HNF4A Gene Increases Fetal Hemoglobin Synthesis in Hudep-2

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

Targeting Transferrin Receptor 2: A Novel "Erythropoiesis-Stimulating Approach" in a Model of Anemia of Chronic Kidney Disease

High-Dose Dexamethasone (HD-DXM) Plus Oseltamivir Versus High-Dose Dexamethasone for Treatment of Adult Primary Immune Thrombocytopenia: Interim Analysis of a Prospective, Multicenter, Randomized, Controlled Trial

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

Platelet and Immune Characteristics of Patients with Immune Thrombocytopaenia Non Responders to Therapeutic Treatments

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

The Translocation t(7;12)(q36;p13) Induces Myeloid Leukemia in Immuno-Compromised but Not Immunocompetent Mice

Recreation of t(7;12)(q36;p13) in Human Induced Pluripotent Stem Cells Using CRISPR/Cas9 Generates a Useful Model for Studying Acute Myeloid Leukemia

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

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

Suicide Gene Therapy with a CD33 Targeted AAV6 Vector Expressing an Inducible Caspase-9 Suicide Gene Is Therapeutic in a Xenotransplantation Model of Acute Myeloid Leukemia

The Factor VIII Variant X5 Enhances Hemophilia a Gene Therapy Efficiency By Its Improved Secretion

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

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