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Journal Articles

Large-scale GMP-compliant CRISPR-Cas9–mediated deletion of the glucocorticoid receptor in multivirus-specific T cells

Rafet Basar, May Daher, Nadima Uprety, Elif Gokdemir, Abdullah Alsuliman, Emily Ensley, Gonca Ozcan, Mayela Mendt, Mayra Hernandez Sanabria, Lucila Nassif Kerbauy, Ana Karen Nunez Cortes, Li Li, Pinaki P. Banerjee, Luis Muniz-Feliciano, Sunil Acharya, Natalie W. Fowlkes, Junjun Lu, Sufang Li, Stephan Mielke, Mecit Kaplan, Vandana Nandivada, Mustafa Bdaiwi, Alexander D. Kontoyiannis, Ye Li, Enli Liu, Sonny Ang, David Marin, Lorenzo Brunetti, Michael C. Gundry, Rolf Turk, Mollie S. Schubert, Garrett R. Rettig, Matthew S. McNeill, Gavin Kurgan, Mark A. Behlke, Richard Champlin, Elizabeth J. Shpall, Katayoun Rezvani
Journal: Blood Advances
Blood Adv (2020) 4 (14): 3357–3367.
https://doi.org/10.1182/bloodadvances.2020001977
Published: 2020
Includes: Supplemental data
Journal Articles

Genome editing of HBG1 and HBG2 to induce fetal hemoglobin

Jean-Yves Métais, Phillip A. Doerfler, Thiyagaraj Mayuranathan, Daniel E. Bauer, Stephanie C. Fowler, Matthew M. Hsieh, Varun Katta, Sagar Keriwala, Cicera R. Lazzarotto, Kevin Luk, Michael D. Neel, S. Scott Perry, Samuel T. Peters, Shaina N. Porter, Byoung Y. Ryu, Akshay Sharma, Devlin Shea, John F. Tisdale, Naoya Uchida, Scot A. Wolfe, Kaitly J. Woodard, Yuxuan Wu, Yu Yao, Jing Zeng, Shondra Pruett-Miller, Shengdar Q. Tsai, Mitchell J. Weiss
Journal: Blood Advances
Blood Adv (2019) 3 (21): 3379–3392.
https://doi.org/10.1182/bloodadvances.2019000820
Published: 2019
Includes: Supplemental data
Journal Articles

High-efficiency CRISPR induction of t(9;11) chromosomal translocations and acute leukemias in human blood stem cells

Johan Jeong, Astraea Jager, Pablo Domizi, Mara Pavel-Dinu, Linda Gojenola, Masayuki Iwasaki, Michael C. Wei, Feng Pan, James L. Zehnder, Matthew H. Porteus, Kara L. Davis, Michael L. Cleary
Journal: Blood Advances
Blood Adv (2019) 3 (19): 2825–2835.
https://doi.org/10.1182/bloodadvances.2019000450
Published: 2019
Includes: Supplemental data
Journal Articles

Fluorescent labeling of CRISPR/Cas9 RNP for gene knockout in HSPCs and iPSCs reveals an essential role for GADD45b in stress response

Masoud Nasri, Perihan Mir, Benjamin Dannenmann, Diana Amend, Tessa Skroblyn, Yun Xu, Klaus Schulze-Osthoff, Maksim Klimiankou, Karl Welte, Julia Skokowa
Journal: Blood Advances
Blood Adv (2019) 3 (1): 63–71.
https://doi.org/10.1182/bloodadvances.2017015511
Published: 2019
Includes: Supplemental data
Journal Articles

Gene editing rescue of a novel MPL mutant associated with congenital amegakaryocytic thrombocytopenia

Cédric Cleyrat, Romain Girard, Eun H. Choi, Éric Jeziorski, Thierry Lavabre-Bertrand, Sylvie Hermouet, Serge Carillo, Bridget S. Wilson
Journal: Blood Advances
Blood Adv (2017) 1 (21): 1815–1826.
https://doi.org/10.1182/bloodadvances.2016002915
Published: 2017
Includes: Supplemental data
Journal Articles

Gene-edited pseudogene resurrection corrects p47 phox -deficient chronic granulomatous disease

Randall K. Merling, Douglas B. Kuhns, Colin L. Sweeney, Xiaolin Wu, Sandra Burkett, Jessica Chu, Janet Lee, Sherry Koontz, Giovanni Di Pasquale, Sandra A. Afione, John A. Chiorini, Elizabeth M. Kang, Uimook Choi, Suk See De Ravin, Harry L. Malech
Journal: Blood Advances
Blood Adv (2016) 1 (4): 270–278.
https://doi.org/10.1182/bloodadvances.2016001214
Published: 2016
Includes: Supplemental data