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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

Leukemia after gene therapy for sickle cell disease: insertional mutagenesis, busulfan, both, or neither

Richard J. Jones, Michael R. DeBaun

Blood (2021) 138 (11): 942–947.

https://doi.org/10.1182/blood.2021011488

Published: 2021

Journal Articles

Introduction to a review series on gene therapy and gene editing for sickle cell disease and hemophilia

Catherine M. Bollard

Blood (2021) 138 (11): 913.

https://doi.org/10.1182/blood.2021012891

Published: 2021

Journal Articles

Gene therapy for hemophilia: a review on clinical benefit, limitations, and remaining issues

Frank W. G. Leebeek, Wolfgang Miesbach

Blood (2021) 138 (11): 923–931.

https://doi.org/10.1182/blood.2019003777

Published: 2021

Journal Articles

A new “FIX” for hemophilia B gene therapy

Graça Almeida-Porada

Blood (2021) 137 (21): 2860–2861.

https://doi.org/10.1182/blood.2021011753

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

von Willebrand disease: what does the future hold?

Cécile V. Denis, Sophie Susen, Peter J. Lenting

Blood (2021) 137 (17): 2299–2306.

https://doi.org/10.1182/blood.2020008501

Published: 2021

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

Diamond-Blackfan anemia

Lydie Da Costa, Thierry Leblanc, Narla Mohandas

Blood (2020) 136 (11): 1262–1273.

https://doi.org/10.1182/blood.2019000947

Published: 2020

Journal Articles

Natural gene therapy in hematopoietic disorders: GATA too

Marjolijn C. Jongmans, Roland P. Kuiper

Blood (2020) 136 (8): 923–924.

https://doi.org/10.1182/blood.2020007001

Published: 2020

Journal Articles

Factor VIII exhibits chaperone-dependent and glucose-regulated reversible amyloid formation in the endoplasmic reticulum

Juthakorn Poothong, Anita Pottekat, Marina Siirin, Alexandre Rosa Campos, Adrienne W. Paton, James C. Paton, Jacqueline Lagunas-Acosta, Zhouji Chen, Mark Swift, Niels Volkmann, Dorit Hanein, Jing Yong, Randal J. Kaufman

Blood (2020) 135 (21): 1899–1911.

https://doi.org/10.1182/blood.2019002867

Published: 2020

Includes: Supplemental data

Journal Articles

The evolution of viral integration site analysis

Stefan Radtke, Hans-Peter Kiem

Blood (2020) 135 (15): 1192–1193.

https://doi.org/10.1182/blood.2020005115

Published: 2020

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

Selecting ß-Thalassemia Patients for Gene Therapy: A Decision-Making Algorithm

Donatella Baronciani, MD, Maddalena Casale, MD, Lucia De Franceschi, MD, Giovanna Graziadei, MD, Filomena Longo, MD, Raffaella Origa, MD, Valeria Maria Pinto, MD, Paolo Rigano, MD, Monia Marchetti, MD, Antonia Gigante, Emanuele Angelucci, MD, Maria Domenica Cappellini, MD, Achille Iolascon, MD PhD, Antonio Piga, Gian Luca Forni

Blood (2019) 134 (Supplement_1): 972.

https://doi.org/10.1182/blood-2019-128320

Published: 2019

Journal Articles

Hemoglobin S Polymerization Can be Inhibited By Alterations in Erythrocyte Glycolysis

William R. Simmons, Quan Li, PhD, John A. Hausman, Suilan Zheng, PhD, William Eaton, MD, Philip S. Low, PhD, David M. Bodine, PhD

Blood (2019) 134 (Supplement_1): 977.

https://doi.org/10.1182/blood-2019-121709

Published: 2019

Journal Articles

Real-World Data of High-Grade Lymphoma Patients Treated with CD19 CAR-T in England

Blood (2019) 134 (Supplement_1): 767.

https://doi.org/10.1182/blood-2019-124177

Published: 2019

Journal Articles

Efficient Correction of ELANE mutations in Primary HSPCs of Severe Congenital Neutropenia Patients Using CRISPR/Cas9 and rAVV6 HDR Repair Templates

Malte U Ritter, Benjamin Secker, Maksim Klimiankou, Masoud Nasri, MSc, Narges Aghaallaei, Benjamin Dannenmann, Regine Bernhard, Ingeborg Steiert, Karin Haehnel, Baubak Bajoghli, Cornelia Zeidler, Karl Welte, Julia Skokowa, MD PhD

Blood (2019) 134 (Supplement_1): 1036.

https://doi.org/10.1182/blood-2019-131708

Published: 2019

Journal Articles

Does Gene Therapy in Beta Thalassemia Normalize Novel Markers of Ineffective Erythropoiesis and Iron Homeostasis?

Alexis A. Thompson, MD MPH, Tomas Ganz, MD PhD, Mary Therese Forsyth, Elizabeta Nemeth, PhD, Sherif M. Badawy, MD

Blood (2019) 134 (Supplement_1): 816.

https://doi.org/10.1182/blood-2019-129658

Published: 2019

NARROW

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

Leukemia after gene therapy for sickle cell disease: insertional mutagenesis, busulfan, both, or neither

Introduction to a review series on gene therapy and gene editing for sickle cell disease and hemophilia

Gene therapy for hemophilia: a review on clinical benefit, limitations, and remaining issues

A new “FIX” for hemophilia B gene therapy

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

von Willebrand disease: what does the future hold?

No CpGs for AAVs?

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

Diamond-Blackfan anemia

Natural gene therapy in hematopoietic disorders: GATA too

Factor VIII exhibits chaperone-dependent and glucose-regulated reversible amyloid formation in the endoplasmic reticulum

The evolution of viral integration site analysis

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

Selecting ß-Thalassemia Patients for Gene Therapy: A Decision-Making Algorithm

Hemoglobin S Polymerization Can be Inhibited By Alterations in Erythrocyte Glycolysis

Real-World Data of High-Grade Lymphoma Patients Treated with CD19 CAR-T in England

Efficient Correction of ELANE mutations in Primary HSPCs of Severe Congenital Neutropenia Patients Using CRISPR/Cas9 and rAVV6 HDR Repair Templates

Does Gene Therapy in Beta Thalassemia Normalize Novel Markers of Ineffective Erythropoiesis and Iron Homeostasis?

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American Society of Hematology

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ASH Publications

American Society of Hematology

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