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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

First-in-human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A

Steven Pipe, MD, Michael Becka, Elke Detering, Konstantina Vanevski, Toshko Lissitchkov

Blood (2019) 134 (Supplement_1): 4630.

https://doi.org/10.1182/blood-2019-125764

Published: 2019

Journal Articles

Clearance of Vector DNA Following Systemic Administration of AAV5-hFIX or AAV5-hFIX Padua in Patients with Severe or Moderate-Severe Hemophilia B

Eileen K Sawyer, PhD, Sander Gielen, PhD, Jaap Twisk, Alison Long, Robert Gut

Blood (2019) 134 (Supplement_1): 2062.

https://doi.org/10.1182/blood-2019-129786

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

A Multistate Survival Analysis for Patients with Follicular Lymphoma (FL) Using 13 First-Line Randomized Trials from FL Analysis of Surrogate Hypothesis (FLASH) Group

Blood (2019) 134 (Supplement_1): 2812.

https://doi.org/10.1182/blood-2019-122328

Published: 2019

Journal Articles

Mutant ASXL1 Disrupts Paraspeckle Formation through Aberrant Interaction with Nono in Hematopoietic Cells

Yamamoto Keita, MD, Susumu Goyama, MD PhD, Shuhei Asada, MD PhD, Takeshi Fujino, MD, Tomofusa Fukuyama, MD PhD, Yosuke Tanaka, MD, Ayana Kon, MD PhD, Yasuhito Nannya, MD PhD, Seishi Ogawa, MD PhD, Reina Onoguchi, PhD, Nobuyoshi Akimitsu, PhD, Toshio Kitamura, MD PhD

Blood (2019) 134 (Supplement_1): 2514.

https://doi.org/10.1182/blood-2019-125472

Published: 2019

Journal Articles

Effective Stabilization of Viral Vectors in Liquid Using an Algorithm-Based Development Approach

Konstantin Petropoulos, PhD

Blood (2019) 134 (Supplement_1): 5752.

https://doi.org/10.1182/blood-2019-130030

Published: 2019

Journal Articles

GFI1-Dependent SGPP1 Repression Promotes Growth and Survival of Myeloma Cells

Daniela Nicoleta Petrusca, PhD, Evgeny Berdyshev, PhD, Colin D. Crean, BS, Judith L Anderson, BS, G. David Roodman, MD PhD

Blood (2019) 134 (Supplement_1): 4387.

https://doi.org/10.1182/blood-2019-131106

Published: 2019

Journal Articles

Activation of JAK2/STAT5 Pathway Reduces Expression Level of DNMT3a in MPN Cell Line

Jie Zhou, Aibin Liang, PhD, Shaoguang Li, Wenjun Zhang, Jianfei FU, MD

Blood (2019) 134 (Supplement_1): 5394.

https://doi.org/10.1182/blood-2019-125434

Published: 2019

Journal Articles

DNA Methylation Profiling of AML Reveals Epigenetic Subgroups with Distinct Clinical Outcome

Sebastian Vosberg, Paul Kerbs, MSc, Vindi Jurinovic, MSc, Klaus H. Metzeler, MD, Susanne Amler, PhD, Cristina Sauerland, Dennis Görlich, PhD, Wolfgang E. Berdel, MD, Jan Braess, MD, Wolfgang Hiddemann, MD PhD, Karsten Spiekermann, MD, Tobias Herold, MD, Philipp A. Greif, MD PD

Blood (2019) 134 (Supplement_1): 2715.

https://doi.org/10.1182/blood-2019-130047

Published: 2019

Journal Articles

Loss of ZBTB7A Enhances Glycolysis and Beta Oxidation in Myeloid Leukemia

Enric Redondo Monte, MSc, BSc, Anja Wilding, MSc, Georg Leubolt, MSc, Paul Kerbs, MSc, Johannes Bagnoli, Wolfgang Hiddemann, MD PhD, Wolfgang Enard, PhD, Sebastian Theurich, MD, Philipp A. Greif, MD PD

Blood (2019) 134 (Supplement_1): 1453.

https://doi.org/10.1182/blood-2019-128378

Published: 2019

Journal Articles

CLL-Derived Exosomes Function As Trojan Horses That Induce SMAD6-Dependent Apoptosis of Normal B-Cells

Orit Uziel, PhD, Zinab Sarsur- Amer, Einat Beery, Pia Raanani, MD, Uri Rozovski, MD

Blood (2019) 134 (Supplement_1): 1734.

https://doi.org/10.1182/blood-2019-129873

Published: 2019

Journal Articles

Dissecting the Immune Cell Progeny of CAR-Expressing Hematopoietic Stem Cells in a Humanized Mouse Model

Heng-Yi Liu, Nezia Rahman, Tzu-Ting Chiou, PhD, Satiro N. De Oliveira, MD

Blood (2019) 134 (Supplement_1): 4640.

https://doi.org/10.1182/blood-2019-129746

Published: 2019

Journal Articles

Foamy Virus Based Vectors for CAR-T Cell Development

Emmanouil Simantirakis, MSc, Vassilis Atsaves, PhD, Ioannis Tsironis, BSc MSc, Margarita Gkyzi, BSc, Kostas Konstantopoulos, MD PhD, George Vassilopoulos, MD PhD

Blood (2019) 134 (Supplement_1): 4646.

https://doi.org/10.1182/blood-2019-123022

Published: 2019

Journal Articles

One Year Data from a Phase 2b Trial of AMT-061 (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B

Steven Pipe, MD, Adam Giermasz, MD PhD, Giancarlo Castaman, MD, Nigel S. Key, MBChB, FRCP, Susan U Lattimore, Frank Leebeek, MD PhD, Wolfgang Miesbach, MD PhD, Michael Recht, MD PhD, Esteban Gomez, MD, Alison Long, Robert Gut, Annette von Drygalski, MD PharmD

Blood (2019) 134 (Supplement_1): 3348.

https://doi.org/10.1182/blood-2019-128765

Published: 2019

Journal Articles

Efficacy and Safety in 15 Hemophilia B Patients Treated with the AAV Gene Therapy Vector Fidanacogene Elaparvovec and Followed for at Least 1 Year

Lindsey A. George, MD, Spencer K. Sullivan, MD, John E.J. Rasko, MBBS, PhD, Adam Giermasz, MD PhD, Benjamin J. Samelson-Jones, MD PhD, Jonathan M. Ducore, MD MPH, Jerome M. Teitel, MD FRCPC, Catherine E. McGuinn, MD, Alexa R. Runowski, MS, Fraser Wright, PhD, Xavier M. Anguela, PhD, Katherine A. High, MD, Denis Rybin, PhD, John E. Murphy, PhD, Jeremy Rupon, MD

Blood (2019) 134 (Supplement_1): 3347.

https://doi.org/10.1182/blood-2019-124091

Published: 2019

Journal Articles

Relapse and Mortality Prediction of Acute Myeloid Leukemia Patients Using Deep Bidirectional Long Short-Term Memory-Deep Neural Network Architecture

Bor-Sheng Ko, MD PhD, Yu-Fen Wang, MS, Chih-Chuan Lu, B.S., Jeng-Lin Li, BS, Chi-Chun Lee, PhD, Jih-Luh Tang, MD, Hwei-Fang Tien, MD, PhD

Blood (2018) 132 (Supplement 1): 2811.

https://doi.org/10.1182/blood-2018-99-115778

Published: 2018

Journal Articles

Synthetic Lethality CRISPR Screens in Isogenic iPSCs with Splicing Factor Gene Mutations

Henrik Sperber, PhD, Shailee Vora, BS, MS, Andriana G. Kotini, PhD, Malgorzata Olszewska, MS, Aleksandra Wroblewska, PhD, Brian Brown, PhD, Eirini P. Papapetrou, MD PhD

Blood (2018) 132 (Supplement 1): 3083.

https://doi.org/10.1182/blood-2018-99-111857

Published: 2018

Journal Articles

SIRT3 Sumoylation Contributes to Chemoresistance in AML

Jiao MA, PhD, Bin Liu, MSc, Dan Yu, BSc, Wayne Tam, MD PhD, Jianmin Yang, PhD, Jinke Cheng, PhD

Blood (2018) 132 (Supplement 1): 3929.

https://doi.org/10.1182/blood-2018-99-110898

Published: 2018

Journal Articles

IGFBP7 As a Potential Therapeutic Target for AML

Yina Niu, PhD, Shuiyan Wu, MD, Shaoyan Hu, MD PhD

Blood (2018) 132 (Supplement 1): 5134.

https://doi.org/10.1182/blood-2018-99-119528

Published: 2018

NARROW

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

First-in-human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A

Clearance of Vector DNA Following Systemic Administration of AAV5-hFIX or AAV5-hFIX Padua in Patients with Severe or Moderate-Severe Hemophilia B

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

A Multistate Survival Analysis for Patients with Follicular Lymphoma (FL) Using 13 First-Line Randomized Trials from FL Analysis of Surrogate Hypothesis (FLASH) Group

Mutant ASXL1 Disrupts Paraspeckle Formation through Aberrant Interaction with Nono in Hematopoietic Cells

Effective Stabilization of Viral Vectors in Liquid Using an Algorithm-Based Development Approach

GFI1-Dependent SGPP1 Repression Promotes Growth and Survival of Myeloma Cells

Activation of JAK2/STAT5 Pathway Reduces Expression Level of DNMT3a in MPN Cell Line

DNA Methylation Profiling of AML Reveals Epigenetic Subgroups with Distinct Clinical Outcome

Loss of ZBTB7A Enhances Glycolysis and Beta Oxidation in Myeloid Leukemia

CLL-Derived Exosomes Function As Trojan Horses That Induce SMAD6-Dependent Apoptosis of Normal B-Cells

Dissecting the Immune Cell Progeny of CAR-Expressing Hematopoietic Stem Cells in a Humanized Mouse Model

Foamy Virus Based Vectors for CAR-T Cell Development

One Year Data from a Phase 2b Trial of AMT-061 (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B

Efficacy and Safety in 15 Hemophilia B Patients Treated with the AAV Gene Therapy Vector Fidanacogene Elaparvovec and Followed for at Least 1 Year

Relapse and Mortality Prediction of Acute Myeloid Leukemia Patients Using Deep Bidirectional Long Short-Term Memory-Deep Neural Network Architecture

Synthetic Lethality CRISPR Screens in Isogenic iPSCs with Splicing Factor Gene Mutations

SIRT3 Sumoylation Contributes to Chemoresistance in AML

IGFBP7 As a Potential Therapeutic Target for AML

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American Society of Hematology

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ASH Publications

American Society of Hematology

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