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Journal Articles

CRISPR/Cas9-mediated in vivo gene targeting corrects hemostasis in newborn and adult factor IX–knockout mice

Lili Wang, Yang Yang, Camilo Ayala Breton, John White, Jia Zhang, Yan Che, Alexei Saveliev, Deirdre McMenamin, Zhenning He, Caitlin Latshaw, Mingyao Li, James M. Wilson
Blood (2019) 133 (26): 2745–2752.
DOI: https://doi.org/10.1182/blood.2019000790
Published: 2019
Journal Articles

CRISPR/Cas9 PIG -A gene editing in nonhuman primate model demonstrates no intrinsic clonal expansion of PNH HSPCs

Tae-Hoon Shin, Eun Jung Baek, Marcus A. F. Corat, Shirley Chen, Jean-Yves Metais, Aisha A. AlJanahi, Yifan Zhou, Robert E. Donahue, Kyung-Rok Yu, Cynthia E. Dunbar
Blood (2019) 133 (23): 2542–2545.
DOI: https://doi.org/10.1182/blood.2019000800
Published: 2019
Journal Articles

Editing aberrant splice sites efficiently restores β-globin expression in β-thalassemia

Brief Report
Shuqian Xu, Kevin Luk, Qiuming Yao, Anne H. Shen, Jing Zeng, Yuxuan Wu, Hong-Yuan Luo, Christian Brendel, Luca Pinello, David H. K. Chui, Scot A. Wolfe, Daniel E. Bauer
Blood (2019) 133 (21): 2255–2262.
DOI: https://doi.org/10.1182/blood-2019-01-895094
Published: 2019
Journal Articles

GM-CSF inhibition reduces cytokine release syndrome and neuroinflammation but enhances CAR-T cell function in xenografts

Rosalie M. Sterner, Reona Sakemura, Michelle J. Cox, Nan Yang, Roman H. Khadka, Cynthia L. Forsman, Michael J. Hansen, Fang Jin, Katayoun Ayasoufi, Mehrdad Hefazi, Kendall J. Schick, Denise K. Walters, Omar Ahmed, Dale Chappell, Tarek Sahmoud, Cameron Durrant, Wendy K. Nevala, Mrinal M. Patnaik, Larry R. Pease, Karen E. Hedin, Neil E. Kay, Aaron J. Johnson, Saad S. Kenderian
Blood (2019) 133 (7): 697–709.
DOI: https://doi.org/10.1182/blood-2018-10-881722
Published: 2019
Journal Articles

Update on clinical gene therapy for hemophilia

Clinical Trials & Observations
George Q. Perrin, Roland W. Herzog, David M. Markusic
Blood (2019) 133 (5): 407–414.
DOI: https://doi.org/10.1182/blood-2018-07-820720
Published: 2019
Journal Articles

Reactivation of γ-globin in adult β-YAC mice after ex vivo and in vivo hematopoietic stem cell genome editing

Chang Li, Nikoletta Psatha, Pavel Sova, Sucheol Gil, Hongjie Wang, Jiho Kim, Chandana Kulkarni, Cristina Valensisi, R. David Hawkins, George Stamatoyannopoulos, André Lieber
Blood (2018) 131 (26): 2915–2928.
DOI: https://doi.org/10.1182/blood-2018-03-838540
Published: 2018
Includes: Supplemental data
Journal Articles

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus

Chiara Antoniani, Vasco Meneghini, Annalisa Lattanzi, Tristan Felix, Oriana Romano, Elisa Magrin, Leslie Weber, Giulia Pavani, Sara El Hoss, Ryo Kurita, Yukio Nakamura, Thomas J. Cradick, Ante S. Lundberg, Matthew Porteus, Mario Amendola, Wassim El Nemer, Marina Cavazzana, Fulvio Mavilio, Annarita Miccio
Blood (2018) 131 (17): 1960–1973.
DOI: https://doi.org/10.1182/blood-2017-10-811505
Published: 2018
Includes: Supplemental data
Journal Articles

Posttransplant chimeric antigen receptor therapy

Melody Smith, Johannes Zakrzewski, Scott James, Michel Sadelain
Blood (2018) 131 (10): 1045–1052.
DOI: https://doi.org/10.1182/blood-2017-08-752121
Published: 2018
Journal Articles

Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B

Clinical Trials & Observations
Wolfgang Miesbach, Karina Meijer, Michiel Coppens, Peter Kampmann, Robert Klamroth, Roger Schutgens, Marco Tangelder, Giancarlo Castaman, Joachim Schwäble, Halvard Bonig, Erhard Seifried, Federica Cattaneo, Christian Meyer, Frank W. G. Leebeek
Blood (2018) 131 (9): 1022–1031.
DOI: https://doi.org/10.1182/blood-2017-09-804419
Published: 2018
Includes: Supplemental data
Journal Articles

Hematopoietic stem cell gene therapy for IFNγR1 deficiency protects mice from mycobacterial infections

Miriam Hetzel, Adele Mucci, Patrick Blank, Ariane Hai Ha Nguyen, Jan Schiller, Olga Halle, Mark-Philipp Kühnel, Sandra Billig, Robert Meineke, Daniel Brand, Vanessa Herder, Wolfgang Baumgärtner, Franz-Christoph Bange, Ralph Goethe, Danny Jonigk, Reinhold Förster, Bernhard Gentner, Jean-Laurent Casanova, Jacinta Bustamante, Axel Schambach, Ulrich Kalinke, Nico Lachmann
Blood (2018) 131 (5): 533–545.
DOI: https://doi.org/10.1182/blood-2017-10-812859
Published: 2018
Includes: Multimedia, Supplemental data
Journal Articles

CRISPR-mediated TCR replacement generates superior anticancer transgenic T cells

Mateusz Legut, Garry Dolton, Afsar Ali Mian, Oliver G. Ottmann, Andrew K. Sewell
Blood (2018) 131 (3): 311–322.
DOI: https://doi.org/10.1182/blood-2017-05-787598
Published: 2018
Includes: Supplemental data
Journal Articles

SLAMF7-CAR T cells eliminate myeloma and confer selective fratricide of SLAMF7 + normal lymphocytes

Tea Gogishvili, Sophia Danhof, Sabrina Prommersberger, Julian Rydzek, Martin Schreder, Christian Brede, Hermann Einsele, Michael Hudecek
Blood (2017) 130 (26): 2838–2847.
DOI: https://doi.org/10.1182/blood-2017-04-778423
Published: 2017
Includes: Supplemental data
Journal Articles

c-MPL provides tumor-targeted T-cell receptor-transgenic T cells with costimulation and cytokine signals

Christopher D. Nishimura, Daniel A. Brenner, Malini Mukherjee, Rachel A. Hirsch, Leah Ott, Meng-Fen Wu, Hao Liu, Olga Dakhova, Jordan S. Orange, Malcolm K. Brenner, Charles Y. Lin, Caroline Arber
Blood (2017) 130 (25): 2739–2749.
DOI: https://doi.org/10.1182/blood-2017-02-769463
Published: 2017
Includes: Supplemental data
Journal Articles

Novel approaches to hemophilia therapy: successes and challenges

Clinical Trials & Observations
Valder R. Arruda, Bhavya S. Doshi, Benjamin J. Samelson-Jones
Blood (2017) 130 (21): 2251–2256.
DOI: https://doi.org/10.1182/blood-2017-08-742312
Published: 2017
Journal Articles

Safety and persistence of WT1-specific T-cell receptor gene−transduced lymphocytes in patients with AML and MDS

Clinical Trials & Observations
Isao Tawara, Shinichi Kageyama, Yoshihiro Miyahara, Hiroshi Fujiwara, Tetsuya Nishida, Yoshiki Akatsuka, Hiroaki Ikeda, Kazushi Tanimoto, Seitaro Terakura, Makoto Murata, Yoko Inaguma, Masahiro Masuya, Naoki Inoue, Tomohide Kidokoro, Sachiko Okamoto, Daisuke Tomura, Hideto Chono, Ikuei Nukaya, Junichi Mineno, Tomoki Naoe, Nobuhiko Emi, Masaki Yasukawa, Naoyuki Katayama, Hiroshi Shiku
Blood (2017) 130 (18): 1985–1994.
DOI: https://doi.org/10.1182/blood-2017-06-791202
Published: 2017
Includes: Supplemental data
Journal Articles

Engraftment and in vivo proliferation advantage of gene-corrected mobilized CD34 + cells from Fanconi anemia patients

Paula Río, Susana Navarro, Guillermo Guenechea, Rebeca Sánchez-Domínguez, Maria Luisa Lamana, Rosa Yañez, Jose A. Casado, Parinda A. Mehta, Maria Roser Pujol, Jordi Surrallés, Sabine Charrier, Anne Galy, José C. Segovia, Cristina Díaz de Heredia, Julián Sevilla, Juan A. Bueren
Blood (2017) 130 (13): 1535–1542.
DOI: https://doi.org/10.1182/blood-2017-03-774174
Published: 2017
Includes: Supplemental data
Journal Articles

Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult

Clinical Trials & Observations
Emma C. Morris, Thomas Fox, Ronjon Chakraverty, Rita Tendeiro, Katie Snell, Christine Rivat, Sarah Grace, Kimberly Gilmour, Sarita Workman, Karen Buckland, Katie Butler, Ronnie Chee, Alan D. Salama, Hazem Ibrahim, Havinder Hara, Cecile Duret, Fulvio Mavilio, Frances Male, Frederick D. Bushman, Anne Galy, Siobhan O. Burns, H. Bobby Gaspar, Adrian J. Thrasher
Blood (2017) 130 (11): 1327–1335.
DOI: https://doi.org/10.1182/blood-2017-04-777136
Published: 2017
Includes: Supplemental data
Journal Articles

NY-ESO-1 TCR single edited stem and central memory T cells to treat multiple myeloma without graft-versus-host disease

Sara Mastaglio, Pietro Genovese, Zulma Magnani, Eliana Ruggiero, Elisa Landoni, Barbara Camisa, Giulia Schiroli, Elena Provasi, Angelo Lombardo, Andreas Reik, Nicoletta Cieri, Martina Rocchi, Giacomo Oliveira, Giulia Escobar, Monica Casucci, Bernhard Gentner, Antonello Spinelli, Anna Mondino, Attilio Bondanza, Luca Vago, Maurilio Ponzoni, Fabio Ciceri, Michael C. Holmes, Luigi Naldini, Chiara Bonini
Blood (2017) 130 (5): 606–618.
DOI: https://doi.org/10.1182/blood-2016-08-732636
Published: 2017
Includes: Supplemental data
Journal Articles

Plasmacytoid and conventional dendritic cells cooperate in crosspriming AAV capsid-specific CD8 + T cells

Geoffrey L. Rogers, Jamie L. Shirley, Irene Zolotukhin, Sandeep R. P. Kumar, Alexandra Sherman, George Q. Perrin, Brad E. Hoffman, Arun Srivastava, Etiena Basner-Tschakarjan, Mark A. Wallet, Cox Terhorst, Moanaro Biswas, Roland W. Herzog
Blood (2017) 129 (24): 3184–3195.
DOI: https://doi.org/10.1182/blood-2016-11-751040
Published: 2017
Includes: Supplemental data
Journal Articles

Cytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients

Aaron R. Cooper, Georgia R. Lill, Kit Shaw, Denise A. Carbonaro-Sarracino, Alejandra Davila, Robert Sokolic, Fabio Candotti, Matteo Pellegrini, Donald B. Kohn
Blood (2017) 129 (19): 2624–2635.
DOI: https://doi.org/10.1182/blood-2016-12-756734
Published: 2017
Includes: Supplemental data