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Journal Articles

CRISPR-targeted MAGT1 insertion restores XMEN patient hematopoietic stem cells and lymphocytes

Blood (2021) 138 (26): 2768–2780.

DOI: https://doi.org/10.1182/blood.2021011192

Published: 2021

Includes: Supplemental data

Journal Articles

Suppression of fibrin(ogen)-driven pathologies through controlled knockdown by lipid nanoparticle delivery of siRNA

Lih Jiin Juang, Woosuk Steve Hur, Lakmali Munasinghage Silva, Amy W Strilchuk, Brenton Francisco, Jerry Leung, Madelaine K. Robertson, Dafna J. Groeneveld, Bridget La Prairie, Elizabeth M. Chun, Andrew P. Cap, James P Luyendyk, Joseph S. Palumbo, Pieter R. Cullis, Thomas H Bugge, Matthew J. Flick, Christian J. Kastrup

Blood blood.2021014559.

DOI: https://doi.org/10.1182/blood.2021014559

Published: 2021

Journal Articles

The cost-effectiveness of gene therapy for severe hemophilia B: a microsimulation study from the United States perspective

Nancy S. Bolous, Yichen Chen, Huiqi Wang, Andrew M. Davidoff, Meenakshi Devidas, Timothy W. Jacobs, Michael M. Meagher, Amit C. Nathwani, Ellis J. Neufeld, Bryan A. Piras, Carlos Rodriguez-Galindo, Ulrike M. Reiss, Nickhill Bhakta

Blood (2021) 138 (18): 1677–1690.

DOI: https://doi.org/10.1182/blood.2021010864

Published: 2021

Includes: Supplemental data

Journal Articles

Enhanced HbF reactivation by multiplex mutagenesis of thalassemic CD34 + cells in vitro and in vivo

Nikoletta Psatha, Aphrodite Georgakopoulou, Chang Li, Vivek Nandakumar, Grigorios Georgolopoulos, Reyes Acosta, Kiriaki Paschoudi, Jemma Nelson, Daniel Chee, Anastasia Athanasiadou, Anastasia Kouvatsi, Alister Peter William Funnell, André Lieber, Evangelia Yannaki, Thalia Papayannopoulou

Blood (2021) 138 (17): 1540–1553.

DOI: https://doi.org/10.1182/blood.2020010020

Published: 2021

Includes: Supplemental data

Journal Articles

Development of CAR T-cell lymphoma in 2 of 10 patients effectively treated with piggyBac -modified CD19 CAR T cells

Blood (2021) 138 (16): 1504–1509.

DOI: https://doi.org/10.1182/blood.2021010813

Published: 2021

Includes: Supplemental data

Journal Articles

Expect the unexpected: piggyBac and lymphoma

Matthew H. Wilson, Stephen Gottschalk

Blood (2021) 138 (16): 1379–1380.

DOI: https://doi.org/10.1182/blood.2021012349

Published: 2021

Journal Articles

Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency

Blood (2021) 138 (15): 1304–1316.

DOI: https://doi.org/10.1182/blood.2020010260

Published: 2021

Includes: Supplemental data

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.

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

Published: 2021

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: a review on clinical benefit, limitations, and remaining issues

Frank W. G. Leebeek, Wolfgang Miesbach

Blood (2021) 138 (11): 923–931.

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

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.

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

Published: 2021

Journal Articles

Interventions and outcomes of adult patients with B-ALL progressing after CD19 chimeric antigen receptor T-cell therapy

Kitsada Wudhikarn, Jessica R. Flynn, Isabelle Rivière, Mithat Gönen, Xiuyan Wang, Brigitte Senechal, Kevin J. Curran, Mikhail Roshal, Peter G. Maslak, Mark B. Geyer, Elizabeth F. Halton, Claudia Diamonte, Marco L. Davila, Michel Sadelain, Renier J. Brentjens, Jae H. Park

Blood (2021) 138 (7): 531–543.

DOI: https://doi.org/10.1182/blood.2020009515

Published: 2021

Includes: Supplemental data

Journal Articles

Prognostic impact of corticosteroids on efficacy of chimeric antigen receptor T-cell therapy in large B-cell lymphoma

Blood (2021) 137 (23): 3272–3276.

DOI: https://doi.org/10.1182/blood.2020008865

Published: 2021

Includes: Supplemental data

Journal Articles

Proof of concept for a rapidly switchable universal CAR-T platform with UniCAR-T-CD123 in relapsed/refractory AML

Martin Wermke, Sabrina Kraus, Armin Ehninger, Ralf C. Bargou, Maria-Elisabeth Goebeler, Jan Moritz Middeke, Carla Kreissig, Malte von Bonin, Jan Koedam, Michael Pehl, Martin Bornhäuser, Hermann Einsele, Gerhard Ehninger, Marc Cartellieri

Blood (2021) 137 (22): 3145–3148.

DOI: https://doi.org/10.1182/blood.2020009759

Published: 2021

Journal Articles

A phase 1 study of a novel fully human BCMA-targeting CAR (CT103A) in patients with relapsed/refractory multiple myeloma

Blood (2021) 137 (21): 2890–2901.

DOI: https://doi.org/10.1182/blood.2020008936

Published: 2021

Includes: Supplemental data

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

Tumor interferon signaling and suppressive myeloid cells are associated with CAR T-cell failure in large B-cell lymphoma

Blood (2021) 137 (19): 2621–2633.

DOI: https://doi.org/10.1182/blood.2020007445

Published: 2021

Includes: Supplemental data

Journal Articles

Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells

Blood (2021) 137 (19): 2598–2608.

DOI: https://doi.org/10.1182/blood.2020008503

Published: 2021

Includes: Supplemental data

Journal Articles

A combination of cyclophosphamide and interleukin-2 allows CD4 + T cells converted to Tregs to control scurfy syndrome

Blood (2021) 137 (17): 2326–2336.

DOI: https://doi.org/10.1182/blood.2020009187

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

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NARROW

CRISPR-targeted MAGT1 insertion restores XMEN patient hematopoietic stem cells and lymphocytes

Suppression of fibrin(ogen)-driven pathologies through controlled knockdown by lipid nanoparticle delivery of siRNA

The cost-effectiveness of gene therapy for severe hemophilia B: a microsimulation study from the United States perspective

Enhanced HbF reactivation by multiplex mutagenesis of thalassemic CD34 + cells in vitro and in vivo

Development of CAR T-cell lymphoma in 2 of 10 patients effectively treated with piggyBac -modified CD19 CAR T cells

Expect the unexpected: piggyBac and lymphoma

Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency

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

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

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

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

Interventions and outcomes of adult patients with B-ALL progressing after CD19 chimeric antigen receptor T-cell therapy

Prognostic impact of corticosteroids on efficacy of chimeric antigen receptor T-cell therapy in large B-cell lymphoma

Proof of concept for a rapidly switchable universal CAR-T platform with UniCAR-T-CD123 in relapsed/refractory AML

A phase 1 study of a novel fully human BCMA-targeting CAR (CT103A) in patients with relapsed/refractory multiple myeloma

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

Tumor interferon signaling and suppressive myeloid cells are associated with CAR T-cell failure in large B-cell lymphoma

Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells

A combination of cyclophosphamide and interleukin-2 allows CD4 + T cells converted to Tregs to control scurfy syndrome

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

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American Society of Hematology

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ASH Publications

American Society of Hematology

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