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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

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

Rationale and Design of a Phase 3 Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

Rodney H Falk, MD, Morie A Gertz, MD, Merrill D Benson, MD, Gustavo Buchele, MD PhD, Michela Brambatti, MD MS, Sotirios Tsimikas, MD, Nicholas J Viney, Bsc, Li-Jung Tai, MD PhD, Cecilia Monteiro, MD PhD, Qingqing Yang, MS, Louis St L O'Dea, MB B.Ch. BAO, FRCP, Ewa Karwatowska-Prokopczuk, MD PhD, Eugene Schneider, MD, Richard S Geary, PhD, Brett P Monia, PhD

Blood (2019) 134 (Supplement_1): 5764.

https://doi.org/10.1182/blood-2019-129269

Published: 2019

Journal Articles

Ferroportin in Erythrocytes: Importance for Iron Homeostasis and its Role in Infection

Tracey Rouault, MD

Blood (2019) 134 (Supplement_1): SCI-27.

https://doi.org/10.1182/blood-2019-121071

Published: 2019

Journal Articles

RhoA Targeting Suppresses Cold-Induced Platelet Lesion and Allows Refrigerated Storage of Functional Human Platelets to Fourteen Days

Shailaja N Hegde, PhD, Huzoor Akbar, PhD, Shawnagay Nestheide, BS, Ashley M Wellendorf, B.S., Breanna Bohan, James F Johnson, Yi Zheng, PhD, Jose A Cancelas, MD

Blood (2019) 134 (Supplement_1): 99.

https://doi.org/10.1182/blood-2019-125359

Published: 2019

Journal Articles

Early Hyperbilirubinemia Is an Independent Predictor of Outcome after Allogeneic Stem Cell Transplantation and Correlates with Markers of Endothelial Cell Dysfunction

Thomas Luft, MD PhD, Hao Dai, Aleksandar Radujkovic, MD, David Schult, Joshua Majer-Lauterbach, Olga Blau, MD PhD, Lars Bullinger, Sihe Jiang, Carsten Müller-Tidow, MD, Peter Dreger, Olaf Penack, MD

Blood (2019) 134 (Supplement_1): 4487.

https://doi.org/10.1182/blood-2019-124284

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

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

The Safety of Novel Drugs Targeting Factor XI and XII in Human Clinical Trials:a Systematic Review

Gillian E Mair, Sven Reid Olson, MD, Joseph J Shatzel, MD

Blood (2019) 134 (Supplement_1): 4966.

https://doi.org/10.1182/blood-2019-130823

Published: 2019

Journal Articles

SIX1 Is a Novel Effector Gene in CALM-AF10 Leukemia

Waitman K. Aumann, MDMS, Catherine P. Lavau, DVM, PhD, Amanda Harrington, MD, Amanda E. Conway, PhD, Daniel S. Wechsler, MD PhD

Blood (2019) 134 (Supplement_1): 5035.

https://doi.org/10.1182/blood-2019-130178

Published: 2019

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

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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

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

Rationale and Design of a Phase 3 Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

Ferroportin in Erythrocytes: Importance for Iron Homeostasis and its Role in Infection

RhoA Targeting Suppresses Cold-Induced Platelet Lesion and Allows Refrigerated Storage of Functional Human Platelets to Fourteen Days

Early Hyperbilirubinemia Is an Independent Predictor of Outcome after Allogeneic Stem Cell Transplantation and Correlates with Markers of Endothelial Cell Dysfunction

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

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

The Safety of Novel Drugs Targeting Factor XI and XII in Human Clinical Trials:a Systematic Review

SIX1 Is a Novel Effector Gene in CALM-AF10 Leukemia

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

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