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Journal Articles

A RUNX1 -FPDMM rhesus macaque model reproduces the human phenotype and predicts challenges to curative gene therapies

Byung-Chul Lee, Yifan Zhou, Erica Bresciani, Neval Ozkaya, Alina Dulau-Florea, Blake Carrington, Tae-Hoon Shin, Valentina Baena, Zulfeqhar A. Syed, So Gun Hong, Tao Zhen, Katherine R. Calvo, Paul Liu, Cynthia E. Dunbar

Blood (2023) 141 (3): 231–237.

DOI: https://doi.org/10.1182/blood.2022018193

Published: 2023

Includes: Supplemental data

Journal Articles

Long-term follow-up of liver-directed, adeno-associated vector-mediated gene therapy in the canine model of hemophilia A

P. Batty, A. M. Mo, D. Hurlbut, J. Ishida, B. Yates, C. Brown, L. Harpell, C. Hough, A. Pender, E. K. Rimmer, S. Sardo Infirri, A. Winterborn, S. Fong, D. Lillicrap

Blood (2022) 140 (25): 2672–2683.

DOI: https://doi.org/10.1182/blood.2021014735

Published: 2022

Includes: Supplemental data

Journal Articles

Cost-effectiveness of polatuzumab vedotin combined with chemoimmunotherapy in untreated diffuse large B-cell lymphoma

Swetha Kambhampati, Monica Saumoy, Yecheskel Schneider, Stacy Pak, Lihua Elizabeth Budde, Matthew G. Mei, Tanya Siddiqi, Leslie L. Popplewell, Yi-Ping Wen, Jasmine Zain, Stephen J. Forman, Larry W. Kwak, Steven T. Rosen, Alexey V. Danilov, Alex F. Herrera, Nikhil R. Thiruvengadam

Blood (2022) 140 (25): 2697–2708.

DOI: https://doi.org/10.1182/blood.2022016624

Published: 2022

Includes: Supplemental data

Journal Articles

Adenine base editor-mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation

Giulia Hardouin, Panagiotis Antoniou, Pierre Martinucci, Tristan Felix, Sandra Manceau, Laure Joseph, Cécile Masson, Samantha Scaramuzza, Giuliana Ferrari, Marina Cavazzana, Annarita Miccio

Blood blood.2022016629.

DOI: https://doi.org/10.1182/blood.2022016629

Published: 2022

Includes: Supplemental data

Journal Articles

NOTCH1 signaling during CD4 + T-cell activation alters transcription factor networks and enhances antigen responsiveness

Alec B. Wilkens, Elena C. Fulton, Margot J. Pont, Gabriel O. Cole, Isabel Leung, Sylvia M. Stull, Matthew R. Hart, Irwin D. Bernstein, Scott N. Furlan, Stanley R. Riddell

Blood (2022) 140 (21): 2261–2275.

DOI: https://doi.org/10.1182/blood.2021015144

Published: 2022

Includes: Supplemental data

Journal Articles

Kruppel-like factor 1–GATA1 fusion protein improves the sickle cell disease phenotype in mice both in vitro and in vivo

Jianqiong Zhu, Hongzhen Li, Wulin Aerbajinai, Chutima Kumkhaek, Mehdi Pirooznia, Ankit Saxena, Pradeep Dagur, Kyung Chin, Griffin P. Rodgers

Blood (2022) 140 (21): 2276–2289.

DOI: https://doi.org/10.1182/blood.2021014877

Published: 2022

Includes: Supplemental data

Journal Articles

Lentiviral Gene Therapy for X-Linked Chronic Granulomatous Disease Recapitulates Endogenous CYBB Regulation and Expression

Ryan L Wong, Sarah Sackey, Devin Brown, Shantha Senadheera, Katelyn Masiuk, Jason P Quintos, Nicole Colindres, Luke Riggan, Richard A Morgan, Harry L Malech, Roger P Hollis, Donald B Kohn

Blood blood.2022016074.

DOI: https://doi.org/10.1182/blood.2022016074

Published: 2022

Includes: Supplemental data

Journal Articles

Genome edited allogeneic donor 'universal' chimeric antigen receptor T Cells

Waseem Qasim

Blood blood.2022016204.

DOI: https://doi.org/10.1182/blood.2022016204

Published: 2022

Journal Articles

Comparative effectiveness of ZUMA-5 (axi-cel) vs SCHOLAR-5 external control in relapsed/refractory follicular lymphoma

Paola Ghione, M. Lia Palomba, Anik R. Patel, Sabela Bobillo, Kevin Deighton, Caron A. Jacobson, Myrna Nahas, Anthony J. Hatswell, A. Scott Jung, Steve Kanters, Julia Thornton Snider, Sattva S. Neelapu, Maria Teresa Ribeiro, M. Alan Brookhart, Herve Ghesquieres, John Radford, John G. Gribben

Blood (2022) 140 (8): 851–860.

DOI: https://doi.org/10.1182/blood.2021014375

Published: 2022

Includes: Supplemental data

Journal Articles

Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC

Geoffrey D. E. Cuvelier, Brent R. Logan, Susan E. Prockop, Rebecca H. Buckley, Caroline Y. Kuo, Linda M. Griffith, Xuerong Liu, Alison Yip, Michael S. Hershfield, Paul G. Ayoub, Theodore B. Moore, Morna J. Dorsey, Richard J. O’Reilly, Neena Kapoor, Sung-Yun Pai, Malika Kapadia, Christen L. Ebens, Lisa R. Forbes Satter, Lauri M. Burroughs, Aleksandra Petrovic, Deepak Chellapandian, Jennifer Heimall, David C. Shyr, Ahmad Rayes, Jeffrey J. Bednarski, Sharat Chandra, Shanmuganathan Chandrakasan, Alfred P. Gillio, Lisa Madden, Troy C. Quigg, Emi H. Caywood, Blachy J. Dávila Saldaña, Kenneth DeSantes, Hesham Eissa, Frederick D. Goldman, Jacob Rozmus, Ami J. Shah, Mark T. Vander Lugt, Monica S. Thakar, Roberta E. Parrott, Caridad Martinez, Jennifer W. Leiding, Troy R. Torgerson, Michael A. Pulsipher, Luigi D. Notarangelo, Morton J. Cowan, Christopher C. Dvorak, Elie Haddad, Jennifer M. Puck, Donald B. Kohn

Blood (2022) 140 (7): 685–705.

DOI: https://doi.org/10.1182/blood.2022016196

Published: 2022

Includes: Supplemental data

Journal Articles

CD19/22 CAR T cells in children and young adults with B-ALL: phase 1 results and development of a novel bicistronic CAR

Blood (2022) 140 (5): 451–463.

DOI: https://doi.org/10.1182/blood.2022015795

Published: 2022

Includes: Supplemental data

Journal Articles

Genetic ablation of PRDM1 in antitumor T cells enhances therapeutic efficacy of adoptive immunotherapy

Toshiaki Yoshikawa, Zhiwen Wu, Satoshi Inoue, Hitomi Kasuya, Hirokazu Matsushita, Yusuke Takahashi, Hiroaki Kuroda, Waki Hosoda, Shiro Suzuki, Yuki Kagoya

Blood (2022) 139 (14): 2156–2172.

DOI: https://doi.org/10.1182/blood.2021012714

Published: 2022

Includes: Supplemental data

Journal Articles

Disrupting the adult globin promoter alleviates promoter competition and reactivates fetal globin gene expression

Sarah K. Topfer, Ruopeng Feng, Peng Huang, Lana C. Ly, Gabriella E. Martyn, Gerd A. Blobel, Mitchell J. Weiss, Kate G. R. Quinlan, Merlin Crossley

Blood (2022) 139 (14): 2107–2118.

DOI: https://doi.org/10.1182/blood.2021014205

Published: 2022

Includes: Supplemental data

Journal Articles

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

Lih Jiin Juang, Woosuk S. Hur, Lakmali M. 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 (2022) 139 (9): 1302–1311.

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

Published: 2022

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

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

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NARROW

A RUNX1 -FPDMM rhesus macaque model reproduces the human phenotype and predicts challenges to curative gene therapies

Long-term follow-up of liver-directed, adeno-associated vector-mediated gene therapy in the canine model of hemophilia A

Cost-effectiveness of polatuzumab vedotin combined with chemoimmunotherapy in untreated diffuse large B-cell lymphoma

Adenine base editor-mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation

NOTCH1 signaling during CD4 + T-cell activation alters transcription factor networks and enhances antigen responsiveness

Kruppel-like factor 1–GATA1 fusion protein improves the sickle cell disease phenotype in mice both in vitro and in vivo

Lentiviral Gene Therapy for X-Linked Chronic Granulomatous Disease Recapitulates Endogenous CYBB Regulation and Expression

Genome edited allogeneic donor 'universal' chimeric antigen receptor T Cells

Comparative effectiveness of ZUMA-5 (axi-cel) vs SCHOLAR-5 external control in relapsed/refractory follicular lymphoma

Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC

CD19/22 CAR T cells in children and young adults with B-ALL: phase 1 results and development of a novel bicistronic CAR

Genetic ablation of PRDM1 in antitumor T cells enhances therapeutic efficacy of adoptive immunotherapy

Disrupting the adult globin promoter alleviates promoter competition and reactivates fetal globin gene expression

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

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

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

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American Society of Hematology

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ASH Publications

American Society of Hematology

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