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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

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

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

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

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

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

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

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

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

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

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

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

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

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

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