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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.

DOI: 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.

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

Published: 2021

Includes: Supplemental data

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.

DOI: https://doi.org/10.1182/blood.2019004625

Published: 2021

Includes: Supplemental data

Journal Articles

No CpGs for AAVs?

Lindsey A. George

Blood (2021) 137 (6): 721–723.

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

Published: 2021

Journal Articles

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

Blood (2020) 135 (15): 1219–1231.

DOI: 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.

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

Published: 2019

Journal Articles

Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia

Angela R. Smith, MD MS, Gary J. Schiller, MD, Gregory M Vercellotti, MD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Erica B. Esrick, MD, David A. Williams, MD, Weston P. Miller, MD, Adrian Woolfson, MD PhD, Mark C. Walters, MD

Blood (2019) 134 (Supplement_1): 3544.

DOI: https://doi.org/10.1182/blood-2019-125743

Published: 2019

Journal Articles

Design and Characterization of FLT210, a Potent Next Generation AAV-hFVIII Vector Candidate

Blood (2019) 134 (Supplement_1): 4638.

DOI: https://doi.org/10.1182/blood-2019-128490

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.

DOI: 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.

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

Published: 2019

Journal Articles

Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study

Janet L. Kwiatkowski, MD MSCE, Alexis A. Thompson, MD MPH, John E.J. Rasko, MBBS, PhD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, MD, Marina Cavazzana, MD PhD, P. Joy Ho, MBBS DPhil FRACP FRCPA, Manfred Schmidt, PhD, Morris Kletzel, MD MBA, Elliott P. Vichinsky, MD, Briana Deary, Ying Chen, PhD, Alexandria Petrusich, Mark C. Walters, MD

Blood (2019) 134 (Supplement_1): 4628.

DOI: https://doi.org/10.1182/blood-2019-125807

Published: 2019

Journal Articles

Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Blood (2019) 134 (Supplement_1): 4629.

DOI: https://doi.org/10.1182/blood-2019-126161

Published: 2019

Journal Articles

Northstar-3: Interim Results from a Phase 3 Study Evaluating Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Either a β0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene

Ashutosh Lal, MD, Franco Locatelli, MD PhD, Janet L. Kwiatkowski, MD MSCE, Andreas E. Kulozik, MD PhD, Evangelia Yannaki, MD, John B. Porter, MA MDFRCP FRCPath, Isabelle Thuret, MD, Martin G. Sauer, MD, Heidi Elliot, Ying Chen, PhD, Richard A. Colvin, MD PhD, Alexis A. Thompson, MD MPH

Blood (2019) 134 (Supplement_1): 815.

DOI: https://doi.org/10.1182/blood-2019-128482

Published: 2019

Journal Articles

Academic, Phase I/II Trial on T Cells Expressing a Second Generation, CD19-Specific Chimeric Antigen Receptor (CAR) and Inducible Caspase 9 Safety Switch for the Treatment of B-Cell Precursor Acute Lymphoblastic Leukemia (BCP-ALL) and B-Cell Non-Hodgkin Lymphoma (B-NHL) in Children

Francesca Del Bufalo, MD, Concetta Quintarelli, PhD, Biagio De Angelis, PhD, Ignazio Caruana, PhD, Matilde Sinibaldi, PhD, Luciana Vinti, MD, Pietro Merli, MD, Mattia Algeri, MD, Annalisa Ruggeri, MD, Federica Galaverna, MD, Valentina Cirillo, PhD, Maria Giuseppina Cefalo, MD, Giuseppina Li Pira, PhD, Giovanna Leone, MD, Valentina Bertaina, PhD, Franca Fassio, PhD, Monica Gunetti, PhD, Stefano Iacovelli, PhD, Franco Locatelli, MD PhD

Blood (2019) 134 (Supplement_1): 1341.

DOI: https://doi.org/10.1182/blood-2019-129821

Published: 2019

Journal Articles

Expression Profiling of mRNA By Next Generation Sequencing and the Development of Algorithm for Predicting Response in Acute Myeloid Leukemia

Maher Albitar, MD, Marina Y Konopleva, MD PhD, Hong Zhang, PhD, Ivan De Dios, BS, Wanlong Ma, Spiraggelos Antzoulatos, Steven M. Kornblau, MD, Antonio Cavazos, Sanam Loghavi, MD, Koichi Takahashi, MD, Hagop M. Kantarjian, MD, Courtney D. DiNardo, MDMSc

Blood (2019) 134 (Supplement_1): 1314.

DOI: https://doi.org/10.1182/blood-2019-127449

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.

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

Published: 2019

Journal Articles

Beyond the Routine CBC: Research CBC Parameters Associated with Myelodysplastic Syndromes and Underlying Mutations

Olga Pozdnyakova, MD PhD, Radu Stefan Niculescu, PhD, Tracey Kroll, BSc, Lisa Golemme, MLS (ASCP), MS, Nolan Raymond, MLS (ASCP), Debra Briggs, MT(ASCP), MS, Annette S. Kim, MD PhD

Blood (2019) 134 (Supplement_1): 5419.

DOI: https://doi.org/10.1182/blood-2019-125654

Published: 2019

Journal Articles

Risk Factors and Outcomes of Patients with Follicular Lymphoma Who Had Histologic Transformation at First Progression after First-Line Immunochemotherapy in the Gallium Study

Carla Casulo, Michael Herold, MD, Wolfgang Hiddemann, MD PhD, Sunil Iyengar, MD, Robert E. Marcus, FRCP, John F. Seymour, MBBS, PhD FRACP, Aino Launonen, MSc, Andrea Knapp, PhD, Tina G Nielsen, MD PhD, Farheen Mir, MBBS, MD

Blood (2019) 134 (Supplement_1): 5268.

DOI: https://doi.org/10.1182/blood-2019-123466

Published: 2019

Journal Articles

BCR-ABL and Src Family Kinases Induce YAP Tyrosine Phosphorylation Resulting in Survivin and Cyclin D1 Expression in Chronic Myeloid Leukemia Cells

Toshiyuki Hori, MD PhD, Kenta Moriyama, MS

Blood (2019) 134 (Supplement_1): 5366.

DOI: https://doi.org/10.1182/blood-2019-124643

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.

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

Published: 2019

2 3 4 5

NARROW

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

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

No CpGs for AAVs?

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

Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia

Design and Characterization of FLT210, a Potent Next Generation AAV-hFVIII Vector Candidate

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

Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study

Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Northstar-3: Interim Results from a Phase 3 Study Evaluating Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Either a β0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene

Academic, Phase I/II Trial on T Cells Expressing a Second Generation, CD19-Specific Chimeric Antigen Receptor (CAR) and Inducible Caspase 9 Safety Switch for the Treatment of B-Cell Precursor Acute Lymphoblastic Leukemia (BCP-ALL) and B-Cell Non-Hodgkin Lymphoma (B-NHL) in Children

Expression Profiling of mRNA By Next Generation Sequencing and the Development of Algorithm for Predicting Response in Acute Myeloid Leukemia

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

Beyond the Routine CBC: Research CBC Parameters Associated with Myelodysplastic Syndromes and Underlying Mutations

Risk Factors and Outcomes of Patients with Follicular Lymphoma Who Had Histologic Transformation at First Progression after First-Line Immunochemotherapy in the Gallium Study

BCR-ABL and Src Family Kinases Induce YAP Tyrosine Phosphorylation Resulting in Survivin and Cyclin D1 Expression in Chronic Myeloid Leukemia Cells

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

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American Society of Hematology

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American Society of Hematology

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