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Journal Articles

Gene therapy for sickle cell disease: moving from the bench to the bedside

Allistair A. Abraham, John F. Tisdale

Blood (2021) 138 (11): 932–941.

https://doi.org/10.1182/blood.2019003776

Published: 2021

Journal Articles

Gene therapy for hemophilia B using CB 2679d-GT: a novel factor IX variant with higher potency than factor IX Padua

Nisha Nair, Dries De Wolf, Phuong Anh Nguyen, Quang Hong Pham, Ermira Samara-Kuko, Jeff Landau, Grant E. Blouse, Marinee K. Chuah, Thierry VandenDriessche

Blood (2021) 137 (21): 2902–2906.

https://doi.org/10.1182/blood.2020006005

Published: 2021

Includes: Supplemental data

Journal Articles

No CpGs for AAVs?

Lindsey A. George

Blood (2021) 137 (6): 721–723.

https://doi.org/10.1182/blood.2020009285

Published: 2021

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

Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs

Blood (2020) 135 (15): 1219–1231.

https://doi.org/10.1182/blood.2019002350

Published: 2020

Includes: Supplemental data

Journal Articles

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

Erica B. Esrick, MD, Maureen Achebe, MD MPH, Myriam Armant, PhD, Pablo Bartolucci, MD PhD, Marioara Felicia Ciuculescu, MD, Heather Daley, Colleen Dansereau, MSN, RN, CPN, Giuseppe Di Caprio, PhD, Bronner Goncalves, MD PhD, Nicolas Hebert, Matthew M Heeney, MD, John M. Higgins, MD PhD, Leslie E. Lehmann, MD, John P Manis, MD, Olivier Negre, PhD, Sarah Nikiforow, MD PhD, Ethan Schonbrun, PhD, David K. Wood, PhD, David A. Williams, MD

Blood (2019) 134 (Supplement_2): LBA-5.

https://doi.org/10.1182/blood-2019-132745

Published: 2019

Journal Articles

Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies

Laura Breda, PhD, Valentina Ghiaccio, PhD, Silvia Pires Lourenco, MSc, Danuta Jadwiga Jarocha, PhD, Yasuhiro Ikawa, MD PhD, Naoto Tanaka, PhD, Virginia Guzikowski, Carla Casu, PhD, Kim Smith-Whitley, MD, Janet L. Kwiatkowski, MD MSCE, Stefano Rivella, PhD

Blood (2019) 134 (Supplement_1): 2242.

https://doi.org/10.1182/blood-2019-124043

Published: 2019

Journal Articles

Aberrant Cathepsin S Induces a Supportive Immune Microenvironment in Follicular Lymphoma

Blood (2019) 134 (Supplement_1): 657.

https://doi.org/10.1182/blood-2019-122663

Published: 2019

Journal Articles

Gata2 Expression and Function in PML-RARA- driven Preleukemia and APL

Casey D.S. Katerndahl, PhD, Timothy J Ley, MD, Michelle A Cai, Timothy P Rooney, Jr., Sai Mukund Ramakrishnan, MS, Sridhar Nonavinkere Srivatsan, MS, Christopher A Miller, PhD

Blood (2019) 134 (Supplement_1): 534.

https://doi.org/10.1182/blood-2019-131883

Published: 2019

Journal Articles

Germline RUNX1 Variation and Predisposition to T-Cell Acute Lymphoblastic Leukemia in Children

Blood (2019) 134 (Supplement_1): 653.

https://doi.org/10.1182/blood-2019-125912

Published: 2019

Journal Articles

Megakaryocyte Ablation Ameliorates Bone Marrow Fibrosis By Reducing Myofibroblast Differentiation in a Mouse Model of Primary Myelofibrosis

Yutein Chung, PhD, Huiyan Jin, PhD, Claire Trasorras, Francina Gonzalez, Sang Hee Min, MD PhD

Blood (2019) 134 (Supplement_1): 2493.

https://doi.org/10.1182/blood-2019-132186

Published: 2019

Journal Articles

AAV8-Specific Immune Adsorption Column: A Treatment Option for Patients with Pre-Existing Anti-AAV8 Neutralizing Antibodies

Anita Kruzik, PhD, Roland Raim, PhD, Dirk Voelkel, PhD, Markus Weiller, PhD, Werner Hoellriegl, PhD, Friedrich Scheiflinger, Hanspeter Rottensteiner, Birgit M Reipert, PhD, Maurus de la Rosa

Blood (2019) 134 (Supplement_1): 5922.

https://doi.org/10.1182/blood-2019-126730

Published: 2019

Journal Articles

Diagnostic Predictive Model for Diagnosis of Heart Failure after Hematopoietic Cell Transplantation (HCT): Comparison of Traditional Statistical with Machine Learning Modeling

Benjamin Djulbegovic, MD, Jennifer Berano Teh, Lennie Wong, PhD, Iztok Hozo, PhD, Saro H. Armenian, DO, MPH

Blood (2019) 134 (Supplement_1): 5799.

https://doi.org/10.1182/blood-2019-130764

Published: 2019

Journal Articles

Prospective Identification of Acute Myeloid Leukemia Patients Who Benefit from Gene-Expression Based Risk Stratification

Diego Chacon, PhD, Ali Braytee, PhD, Yizhou Huang, PhD, Julie Thoms, PhD, Shruthi Subramanian, BSc, Maria Cristina Sauerland, Stefan K. Bohlander, MD, Jan Braess, MD, Bernhard J. Wörmann, MD, Wolfgang E. Berdel, MD, Wolfgang Hiddemann, MD PhD, Bogdan Gabrys, PhD, Klaus H. Metzeler, MD, Tobias Herold, MD, John Pimanda, MBBS, PhD, Dominik Beck, PhD

Blood (2019) 134 (Supplement_1): 1397.

https://doi.org/10.1182/blood-2019-129519

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

C/EBPβ Isoforms Regulate Proliferation and Differentiation of Regenerating Hematopoietic Stem/Progenitor Cells

Atsushi Sato, MD, Asumi Yokota, PhD, Yoshihiro Hayashi, MD PhD, Naoka Kamio, MD, Satoshi Sagai, Taira Maekawa, MD PhD, Hideyo Hirai, MD PhD

Blood (2019) 134 (Supplement_1): 3713.

https://doi.org/10.1182/blood-2019-128026

Published: 2019

Journal Articles

Next Generation NKG2D-based CAR T-cells (CYAD-02): Co-expression of a Single shRNA Targeting MICA and MICB Improves Cell Persistence and Anti-Tumor Efficacy in vivo

Martina Fontaine, PhD, Benjamin Demoulin, PhD, Simon Bornschein, PhD, Susanna Raitano, PhD, Steve Lenger, Hidevaldo Machado, Jonathan D Moore, Panagiota A. Sotiropoulou, PhD, David E Gilham, PhD

Blood (2019) 134 (Supplement_1): 3931.

https://doi.org/10.1182/blood-2019-129998

Published: 2019

Journal Articles

Identification of T-Cell Receptors Specific to Antigens Presented By HLA-B4002 and B5401 in Acquired Aplastic Anemia

Kohei Hosokawa, MD PhD, Eiji Kobayashi, PhD, Yoshiki Akatsuka, MD PhD, Luis Espinoza, MD PhD, Noriharu Nakagawa, MD, Tanabe Mikoto, MD, Noriaki Tsuji, MD, Takeshi Yoroidaka, MD, Hiroki Mizumaki, MD, Thi Mai Anh Nguyen, MD, Takamasa Katagiri, PhD, Kiyomi Shitaoka, PhD, Hiroshi Hamana, PhD, Hiroyuki Kishi, PhD, Shinji Nakao, MD PhD

Blood (2019) 134 (Supplement_1): 3751.

https://doi.org/10.1182/blood-2019-125685

Published: 2019

Journal Articles

NUP98-HOXA9 -Initiates Molecular and Biologic Features of Disease Progression in a Humanized Model of CML

Ivan Sloma, PharmD, PhD, Philip Beer, PhD FRCPath, MRCP, Christophe Desterke, PhD, Elizabeth Bulaeva, Misha Bilenky, Michelle Moksa, Kamini Raghuram, Cedric Brimacombe, Karen Lambie, Ali G Turhan, MD PhD, Orianne Wagner-Ballon, MD PhD, Philippe Gaulard, MD PhD, R. Keith Humphries, MD PhD, Martin Hirst, PhD BS, Connie J. Eaves, PhD

Blood (2019) 134 (Supplement_1): 4139.

https://doi.org/10.1182/blood-2019-127839

Published: 2019

Journal Articles

Overcoming CD19 Antigen Loss in B-Cell Malignancies with CAR T Cells Targeting BAFF-R

Hong Qin, MD PhD, Zhenyuan Dong, PhD, Xiuli Wang, PhD, Wesley Cheng, BS, Diane Lynne Smith, PhD, Joo Y Song, MD, Ibrahim Aldoss, MD, Stephen J Forman, MD, Larry W Kwak, MD PhD

Blood (2019) 134 (Supplement_1): 3871.

https://doi.org/10.1182/blood-2019-129228

Published: 2019

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Gene therapy for sickle cell disease: moving from the bench to the bedside

Gene therapy for hemophilia B using CB 2679d-GT: a novel factor IX variant with higher potency than factor IX Padua

No CpGs for AAVs?

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

Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies

Aberrant Cathepsin S Induces a Supportive Immune Microenvironment in Follicular Lymphoma

Gata2 Expression and Function in PML-RARA- driven Preleukemia and APL

Germline RUNX1 Variation and Predisposition to T-Cell Acute Lymphoblastic Leukemia in Children

Megakaryocyte Ablation Ameliorates Bone Marrow Fibrosis By Reducing Myofibroblast Differentiation in a Mouse Model of Primary Myelofibrosis

AAV8-Specific Immune Adsorption Column: A Treatment Option for Patients with Pre-Existing Anti-AAV8 Neutralizing Antibodies

Diagnostic Predictive Model for Diagnosis of Heart Failure after Hematopoietic Cell Transplantation (HCT): Comparison of Traditional Statistical with Machine Learning Modeling

Prospective Identification of Acute Myeloid Leukemia Patients Who Benefit from Gene-Expression Based Risk Stratification

Loss of ZBTB7A Enhances Glycolysis and Beta Oxidation in Myeloid Leukemia

C/EBPβ Isoforms Regulate Proliferation and Differentiation of Regenerating Hematopoietic Stem/Progenitor Cells

Next Generation NKG2D-based CAR T-cells (CYAD-02): Co-expression of a Single shRNA Targeting MICA and MICB Improves Cell Persistence and Anti-Tumor Efficacy in vivo

Identification of T-Cell Receptors Specific to Antigens Presented By HLA-B4002 and B5401 in Acquired Aplastic Anemia

NUP98-HOXA9 -Initiates Molecular and Biologic Features of Disease Progression in a Humanized Model of CML

Overcoming CD19 Antigen Loss in B-Cell Malignancies with CAR T Cells Targeting BAFF-R

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American Society of Hematology

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