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Journal Articles

Enhanced self-renewal of human long-term hematopoietic stem cells by a sulfamoyl benzoate derivative targeting p18 INK4C

Blood Adv (2021) 5 (17): 3362–3372.

https://doi.org/10.1182/bloodadvances.2020004054

Published: 2021

Includes: Supplemental data

Journal Articles

Lentiviral correction of enzymatic activity restrains macrophage inflammation in adenosine deaminase 2 deficiency

Blood Adv (2021) 5 (16): 3174–3187.

https://doi.org/10.1182/bloodadvances.2020003811

Published: 2021

Includes: Supplemental data

Journal Articles

Wild-type HIV infection after treatment with lentiviral gene therapy for β-thalassemia

Suradej Hongeng, Usanarat Anurathapan, Duantida Songdej, Angsana Phuphuakrat, Kesinee Jongrak, Geoffrey Parsons, Briana Deary, Melissa Bonner, Gabor Veres, Mohammed Asmal

Blood Adv (2021) 5 (13): 2701–2706.

https://doi.org/10.1182/bloodadvances.2020003680

Published: 2021

Journal Articles

Disease severity impacts plerixafor-mobilized stem cell collection in patients with sickle cell disease

Alexis Leonard, Akshay Sharma, Naoya Uchida, David Stroncek, Sandhya R. Panch, Kamille West, Eoghan Molloy, Thomas E. Hughes, Sara Hauffe, Tiffani Taylor, Courtney Fitzhugh, Jane S. Hankins, Megan Wilson, Shengdar Q. Tsai, Mitchell J. Weiss, Matthew Hsieh, John F. Tisdale

Blood Adv (2021) 5 (9): 2403–2411.

https://doi.org/10.1182/bloodadvances.2021004232

Published: 2021

Includes: Supplemental data

Journal Articles

Immunotherapy perspectives in the new era of B-cell editing

Natsuko Ueda, Marine Cahen, Yannic Danger, Jérôme Moreaux, Christophe Sirac, Michel Cogné

Blood Adv (2021) 5 (6): 1770–1779.

https://doi.org/10.1182/bloodadvances.2020003792

Published: 2021

Journal Articles

Evolutionary insights into coagulation factor IX Padua and other high-specific-activity variants

Benjamin J. Samelson-Jones, Jonathan D. Finn, Leslie J. Raffini, Elizabeth P. Merricks, Rodney M. Camire, Timothy C. Nichols, Valder R. Arruda

Blood Adv (2021) 5 (5): 1324–1332.

https://doi.org/10.1182/bloodadvances.2019000405

Published: 2021

Includes: Supplemental data

Journal Articles

Platelet-targeted hyperfunctional FIX gene therapy for hemophilia B mice even with preexisting anti-FIX immunity

Jocelyn A. Schroeder, Juan Chen, Yingyu Chen, Yuanhua Cai, Hongyin Yu, Jeremy G. Mattson, Paul E. Monahan, Qizhen Shi

Blood Adv (2021) 5 (5): 1224–1238.

https://doi.org/10.1182/bloodadvances.2020004071

Published: 2021

Includes: Supplemental data

Journal Articles

Single-dose MGTA-145/plerixafor leads to efficient mobilization and in vivo transduction of HSCs with thalassemia correction in mice

Chang Li, Kevin A. Goncalves, Tamás Raskó, Amit Pande, Sucheol Gil, Zhinan Liu, Zsuzsanna Izsvák, Thalia Papayannopoulou, John C. Davis, Jr, Hans-Peter Kiem, André Lieber

Blood Adv (2021) 5 (5): 1239–1249.

https://doi.org/10.1182/bloodadvances.2020003714

Published: 2021

Includes: Supplemental data

Journal Articles

In vivo HSPC gene therapy with base editors allows for efficient reactivation of fetal γ-globin in β-YAC mice

Chang Li, Aphrodite Georgakopoulou, Arpit Mishra, Sucheol Gil, R. David Hawkins, Evangelia Yannaki, André Lieber

Blood Adv (2021) 5 (4): 1122–1135.

https://doi.org/10.1182/bloodadvances.2020003702

Published: 2021

Includes: Supplemental data

Journal Articles

Gene therapy for hemophilia: anticipating the unexpected

Glenn F. Pierce

Blood Adv (2020) 4 (15): 3788.

https://doi.org/10.1182/bloodadvances.2020002142

Published: 2020

Includes: Supplemental data

Journal Articles

Curative vs targeted therapy for SCD: does it make more sense to address the root cause than target downstream events?

Marilyn J. Telen

Blood Adv (2020) 4 (14): 3457–3465.

https://doi.org/10.1182/bloodadvances.2020001469

Published: 2020

Journal Articles

Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease

Matthew M. Hsieh, Melissa Bonner, Francis John Pierciey, Jr, Naoya Uchida, James Rottman, Laura Demopoulos, Manfred Schmidt, Julie Kanter, Mark C. Walters, Alexis A. Thompson, Mohammed Asmal, John F. Tisdale

Blood Adv (2020) 4 (9): 2058–2063.

https://doi.org/10.1182/bloodadvances.2019001330

Published: 2020

Journal Articles

High-level protein production in erythroid cells derived from in vivo transduced hematopoietic stem cells

Hongjie Wang, Zhinan Liu, Chang Li, Sucheol Gil, Thalia Papayannopoulou, Christopher B. Doering, André Lieber

Blood Adv (2019) 3 (19): 2883–2894.

https://doi.org/10.1182/bloodadvances.2019000706

Published: 2019

Includes: Supplemental data

Journal Articles

Nongenotoxic antibody-drug conjugate conditioning enables safe and effective platelet gene therapy of hemophilia A mice

Chunyan Gao, Jocelyn A. Schroeder, Feng Xue, Weiqing Jing, Yuanhua Cai, Amelia Scheck, Saravanan Subramaniam, Sridhar Rao, Hartmut Weiler, Agnieszka Czechowicz, Qizhen Shi

Blood Adv (2019) 3 (18): 2700–2711.

https://doi.org/10.1182/bloodadvances.2019000516

Published: 2019

Includes: Supplemental data

Journal Articles

Immunoadsorption enables successful rAAV5-mediated repeated hepatic gene delivery in nonhuman primates

David Salas, Karin L. Kwikkers, Nerea Zabaleta, Andrea Bazo, Harald Petry, Sander J. van Deventer, Gloria Gonzalez Aseguinolaza, Valerie Ferreira

Blood Adv (2019) 3 (17): 2632–2641.

https://doi.org/10.1182/bloodadvances.2019000380

Published: 2019

Includes: Supplemental data

Journal Articles

In vivo dynamics of human hematopoietic stem cells: novel concepts and future directions

Serena Scala, Alessandro Aiuti

Blood Adv (2019) 3 (12): 1916–1924.

https://doi.org/10.1182/bloodadvances.2019000039

Published: 2019

Journal Articles

Baboon envelope LVs efficiently transduced human adult, fetal, and progenitor T cells and corrected SCID-X1 T-cell deficiency

Ornellie Bernadin, Fouzia Amirache, Anais Girard-Gagnepain, Ranjita Devi Moirangthem, Camille Lévy, Kuiying Ma, Caroline Costa, Didier Nègre, Christian Reimann, David Fenard, Agata Cieslak, Vahid Asnafi, Hanem Sadek, Rana Mhaidly, Marina Cavazzana, Chantal Lagresle-Peyrou, François-Loïc Cosset, Isabelle André, Els Verhoeyen

Blood Adv (2019) 3 (3): 461–475.

https://doi.org/10.1182/bloodadvances.2018027508

Published: 2019

Includes: Supplemental data

Journal Articles

Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients

Erica B. Esrick, John P. Manis, Heather Daley, Cristina Baricordi, Hélène Trébéden-Negre, Francis J. Pierciey, Myriam Armant, Sarah Nikiforow, Matthew M. Heeney, Wendy B. London, Luca Biasco, Mohammed Asmal, David A. Williams, Alessandra Biffi

Blood Adv (2018) 2 (19): 2505–2512.

https://doi.org/10.1182/bloodadvances.2018016725

Published: 2018

Includes: Supplemental data

Journal Articles

Gene therapy in hemophilia A: a cost-effectiveness analysis

Nicoletta Machin, Margaret V. Ragni, Kenneth J. Smith

Blood Adv (2018) 2 (14): 1792–1798.

https://doi.org/10.1182/bloodadvances.2018021345

Published: 2018

Journal Articles

Rapid immune reconstitution of SCID-X1 canines after G-CSF/AMD3100 mobilization and in vivo gene therapy

Olivier Humbert, Frieda Chan, Yogendra S. Rajawat, Troy R. Torgerson, Christopher R. Burtner, Nicholas W. Hubbard, Daniel Humphrys, Zachary K. Norgaard, Patricia O’Donnell, Jennifer E. Adair, Grant D. Trobridge, Andrew M. Scharenberg, Peter J. Felsburg, David J. Rawlings, Hans-Peter Kiem

Blood Adv (2018) 2 (9): 987–999.

https://doi.org/10.1182/bloodadvances.2018016451

Published: 2018

Includes: Supplemental data

NARROW

Enhanced self-renewal of human long-term hematopoietic stem cells by a sulfamoyl benzoate derivative targeting p18 INK4C

Lentiviral correction of enzymatic activity restrains macrophage inflammation in adenosine deaminase 2 deficiency

Wild-type HIV infection after treatment with lentiviral gene therapy for β-thalassemia

Disease severity impacts plerixafor-mobilized stem cell collection in patients with sickle cell disease

Immunotherapy perspectives in the new era of B-cell editing

Evolutionary insights into coagulation factor IX Padua and other high-specific-activity variants

Platelet-targeted hyperfunctional FIX gene therapy for hemophilia B mice even with preexisting anti-FIX immunity

Single-dose MGTA-145/plerixafor leads to efficient mobilization and in vivo transduction of HSCs with thalassemia correction in mice

In vivo HSPC gene therapy with base editors allows for efficient reactivation of fetal γ-globin in β-YAC mice

Gene therapy for hemophilia: anticipating the unexpected

Curative vs targeted therapy for SCD: does it make more sense to address the root cause than target downstream events?

Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease

High-level protein production in erythroid cells derived from in vivo transduced hematopoietic stem cells

Nongenotoxic antibody-drug conjugate conditioning enables safe and effective platelet gene therapy of hemophilia A mice

Immunoadsorption enables successful rAAV5-mediated repeated hepatic gene delivery in nonhuman primates

In vivo dynamics of human hematopoietic stem cells: novel concepts and future directions

Baboon envelope LVs efficiently transduced human adult, fetal, and progenitor T cells and corrected SCID-X1 T-cell deficiency

Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients

Gene therapy in hemophilia A: a cost-effectiveness analysis

Rapid immune reconstitution of SCID-X1 canines after G-CSF/AMD3100 mobilization and in vivo gene therapy

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American Society of Hematology

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ASH Publications

American Society of Hematology

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