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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
Journal: Blood Advances
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
Journal: Blood Advances
Blood Adv (2021) 5 (4): 1122–1135.
https://doi.org/10.1182/bloodadvances.2020003702
Published: 2021
Includes: Supplemental data
Journal Articles

Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B

Annette Von Drygalski, Adam Giermasz, Giancarlo Castaman, Nigel S. Key, Susan Lattimore, Frank W. G. Leebeek, Wolfgang Miesbach, Michael Recht, Alison Long, Robert Gut, Eileen K. Sawyer, Steven W. Pipe
Journal: Blood Advances
Blood Adv (2019) 3 (21): 3241–3247.
https://doi.org/10.1182/bloodadvances.2019000811
Published: 2019
Includes: Supplemental data
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
Journal: Blood Advances
Blood Adv (2019) 3 (19): 2883–2894.
https://doi.org/10.1182/bloodadvances.2019000706
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
Journal: Blood Advances
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
Journal: Blood Advances
Blood Adv (2019) 3 (12): 1916–1924.
https://doi.org/10.1182/bloodadvances.2019000039
Published: 2019
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
Journal: Blood Advances
Blood Adv (2018) 2 (9): 987–999.
https://doi.org/10.1182/bloodadvances.2018016451
Published: 2018
Includes: Supplemental data
Journal Articles

Complete correction of hemophilia B phenotype by FIX-Padua skeletal muscle gene therapy in an inhibitor-prone dog model

Robert A. French, Benjamin J. Samelson-Jones, Glenn P. Niemeyer, Clinton D. Lothrop, Jr, Elizabeth P. Merricks, Timothy C. Nichols, Valder R. Arruda
Journal: Blood Advances
Blood Adv (2018) 2 (5): 505–508.
https://doi.org/10.1182/bloodadvances.2017015313
Published: 2018
Includes: Supplemental data
Journal Articles

Hemophilia gene therapy comes of age

Lindsey A. George
Journal: Blood Advances
Blood Adv (2017) 1 (26): 2591–2599.
https://doi.org/10.1182/bloodadvances.2017009878
Published: 2017
Journal Articles

Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors

Amine Meliani, Florence Boisgerault, Zachary Fitzpatrick, Solenne Marmier, Christian Leborgne, Fanny Collaud, Marcelo Simon Sola, Severine Charles, Giuseppe Ronzitti, Alban Vignaud, Laetitia van Wittenberghe, Beatrice Marolleau, Fabienne Jouen, Sisareuth Tan, Olivier Boyer, Olivier Christophe, Alain R. Brisson, Casey A. Maguire, Federico Mingozzi
Journal: Blood Advances
Blood Adv (2017) 1 (23): 2019–2031.
https://doi.org/10.1182/bloodadvances.2017010181
Published: 2017
Includes: Supplemental data