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Journal Articles

Therapeutic Gene Editing of HSCs Ex Vivo without in Vitro Culture Avoids Genotoxicity, Simplifies Procedures, and Preserves Efficiency and Stemness

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Jing Zeng, My Anh Nguyen, Marioara-Felicia Ciuculescu, Esther Mintzer, Pengpeng Liu, Linda Yingqi Lin, Tolulope O Rosanwo, Archana Verma, Nola Neri, Stacy A. Maitland, Alden Richter, David G. Justus, Kendell Clement, Christian Brendel, Luca Pinello, John P Manis, Myriam Armant, Scot A. Wolfe, Daniel E. Bauer
Journal: Blood
Blood (2022) 140 (Supplement 1): 10671–10672.
https://doi.org/10.1182/blood-2022-169900
Published: 2022
Journal Articles

Targeting a Putative Intronic Splicing Silencer Salvages Expression from the Recurrent SBDS C.258+2T>C Mutant Allele in Shwachman-Diamond Syndrome Patient Cells and Mouse Model

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Michael J. Peters, Anna Deck, April Hu, Yuko Fujiwara, Jinkuk Kim, Aubrie Soucy, Kevin Zhang, Julianna Kenny, Linda Y. Lin, Alyssa L. Kennedy, Caitlin Bowers, Chad E. Harris, Chi Yuan Zhang, Karthik Ponnienselvan, Pengpeng Liu, Kevin Luk, Davide Seruggia, R. Coleman Lindsley, John P. Manis, Christian Brendel, Scot A. Wolfe, Stuart H. Orkin, Timothy Yu, Akiko Shimamura, Daniel E. Bauer
Journal: Blood
Blood (2022) 140 (Supplement 1): 2944–2945.
https://doi.org/10.1182/blood-2022-169379
Published: 2022
Journal Articles

Combined +58 and +55 BCL11A enhancer Editing Yields Exceptional Efficiency, Specificity and HbF Induction in Human and NHP Preclinical Models

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Jing Zeng, Selami Demirci, My Anh Nguyen, Linda Yingqi Lin, Stacy A. Maitland, Esther Mintzer, Yuxuan Wu, Danilo Pellin, Amornrat Tangprasittipap, Chokdee Vong, Shaina N. Porter, Kevin Luk, Pengpeng Liu, Varun Katta, Marioara-Felicia Ciuculescu, Daniela Abriss, Jonathan Hsu, Naoya Uchida, Khaled Essawi, Robert Donahue, Karl Petri, Vikram Pattanayak, Luca Pinello, Christian Brendel, David A Williams, John P Manis, Shengdar Q. Tsai, Shondra M. Pruett-Miller, J. Keith Joung, Duantida Songdej, Suradej Hongeng, Myriam Armant, Scot A. Wolfe, John F. Tisdale, Daniel E. Bauer
Journal: Blood
Blood (2021) 138 (Supplement 1): 1852.
https://doi.org/10.1182/blood-2021-152634
Published: 2021
Journal Articles

Nivolumab treatment of relapsed/refractory Epstein-Barr virus–associated hemophagocytic lymphohistiocytosis in adults

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Clinical Trials & Observations
Pengpeng Liu, Xiangyu Pan, Chong Chen, Ting Niu, Xiao Shuai, Jian Wang, Xuelan Chen, Jiazhuo Liu, Yong Guo, Liping Xie, Yu Wu, Yu Liu, Ting Liu
Journal: Blood
Blood (2020) 135 (11): 826–833.
https://doi.org/10.1182/blood.2019003886
Published: 2020
Includes: Supplemental data
Journal Articles

Alox15b Gene Contributes to Lymphoma Tumorigenesis Via PI3K/AKT/mTOR Pathway Activation and Has a Synergistic Effect with Alox5 Gene

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Pengpeng Liu, MD, Yu Liu, PhD, Chong Chen, PhD, Ting Liu, MD, Ting Niu, MD PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 4112.
https://doi.org/10.1182/blood-2018-99-120130
Published: 2018
Journal Articles

Highly Efficient Therapeutic Gene Editing of BCL11A enhancer in Human Hematopoietic Stem Cells from ß-Hemoglobinopathy Patients for Fetal Hemoglobin Induction

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Jing Zeng, MD, Daniel E. Bauer, MD PhD, Yuxuan Wu, PhD, Benjamin P Roscoe, Pengpeng Liu, Qiuming Yao, PhD, Cicera Lazzarrotto, M. Kendell Clement, Mitchel A. Cole, BS, Kevin Luk, Cristina Baricordi, Erica B. Esrick, MD, John P Manis, MD, David Dorfma, David A. Williams, MD, Alessandra Biffi, MD PhD, Carlo Brugnara, MD, Luca Biasco, Christian Brendel, PhD, Luca Pinello, PhD, Shengdar Tsai, PhD, Scot A. Wolfe
Journal: Blood
Blood (2018) 132 (Supplement 1): 3482.
https://doi.org/10.1182/blood-2018-99-119365
Published: 2018