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Shengdar Q. Tsai
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Journal Articles

Systematic Mapping of Gene-Editable Mutations in GATA2 and SAMD9/SAMD9L Syndromes

Damian Krzyzanowski, Sushree S Sahoo, Lili Kotmayer, Masanori Yoshida, Majd Khiami, Alessandra Giorgetti, Shengdar Q Tsai, Senthil V Bhoopalan, Jonathan S Yen, Marcin W Wlodarski
Journal: Blood
Blood (2023) 142 (Supplement 1): 1362.
https://doi.org/10.1182/blood-2023-190982
Published: 2023
Journal Articles

CRISPR-targeted MAGT1 insertion restores XMEN patient hematopoietic stem cells and lymphocytes

Julie Brault, Taylor Liu, Ezekiel Bello, Siyuan Liu, Colin L. Sweeney, Ronald J. Meis, Sherry Koontz, Cristina Corsino, Uimook Choi, Guillaume Vayssiere, Marita Bosticardo, Kennichi Dowdell, Cicera R. Lazzarotto, Aaron B. Clark, Luigi D. Notarangelo, Juan C. Ravell, Michael J. Lenardo, Benjamin P. Kleinstiver, Shengdar Q. Tsai, Xiaolin Wu, Gary A. Dahl, Harry L. Malech, Suk See De Ravin
Journal: Blood
Blood (2021) 138 (26): 2768–2780.
https://doi.org/10.1182/blood.2021011192
Published: 2021
Includes: Supplemental data
Journal Articles

Human Genetic Diversity Alters Therapeutic Gene Editing Off-Target Outcomes

Linda Yingqi Lin, Samuele Cancellieri, Jing Zeng, Francesco Masillo, My Anh Nguyen, Nicola Bombieri, Stacy A. Maitland, Marioara-Felicia Ciuculescu, Varun Katta, Shengdar Q. Tsai, Myriam Armant, Scot A. Wolfe, Rosalba Giugno, Luca Pinello, Daniel E. Bauer
Journal: Blood
Blood (2021) 138 (Supplement 1): 3993.
https://doi.org/10.1182/blood-2021-152429
Published: 2021
Journal Articles

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

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

Adenosine Base Editing of γ-Globin Promoters Induces Fetal Hemoglobin and Inhibit Erythroid Sickling

Thiyagaraj Mayuranathan, Jonathan S. Yen, PhD, Gregory A. Newby, PhD, Yu Yao, MD, Shaina N. Porter, PhD, Kaitly J. Woodard, BS, Jingjing Zhang, MS, Kalin Mayberry, BS, Akshay Sharma, MBBS, Shondra M. preutt-Miller, PhD, John F. Tisdale, MD, Shengdar Q. Tsai, PhD, David R. Liu, PhD, Mitchell J. Weiss, MD PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 21–22.
https://doi.org/10.1182/blood-2020-141498
Published: 2020
Journal Articles

Base Editing Eliminates the Sickle Cell Mutation and Pathology in Hematopoietic Stem Cells Derived Erythroid Cells

Jonathan S Yen, PhD, Gregory A. Newby, PhD, Thiyagaraj Mayuranathan, Shaina N. Porter, PhD, Yu Yao, MD, Kaitly J. Woodard, BS, Kalin Mayberry, BS, Kelcee Everette, Jingjing Zhang, MS, Jordana M Henderson, MS, Anton P Mccaffrey, Jonathan Yen, John F. Tisdale, MD, Shengdar Q. Tsai, PhD, David R. Liu, PhD, Mitchell J. Weiss, MD PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 13–14.
https://doi.org/10.1182/blood-2020-139016
Published: 2020
Journal Articles

Durable and Robust Fetal Globin Induction without Anemia in Rhesus Monkeys Following Autologous Hematopoietic Stem Cell Transplant with BCL11A Erythroid Enhancer Editing

Selami Demirci, PhD, Jing Zeng, MD, Yuxuan Wu, PhD, Naoya Uchida, MD, Jackson Gamer, Morgan Yapundich, Claire Drysdale, Aylin C. Bonifacino, Allen E. Krouse, Nathaniel S. Linde, Robert E. Donahue, VMD, Anne H. Shen, Juan J. Haro-Mora, PhD, Alexis Leonard, MD, Tina Nassehi, Kevin Luk, Scot A. Wolfe, Cicera R. Lazzarotto, Shengdar Q. Tsai, PhD, Mitchell J. Weiss, MD, PhD, Daniel E. Bauer, MD PhD, John F. Tisdale, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4632.
https://doi.org/10.1182/blood-2019-129394
Published: 2019
Journal Articles

CRISPR-Cas9 Genome Editing of γ-Globin Promoters in Human Hematopoietic Stem Cells to Induce Erythrocyte Fetal Hemoglobin for Treatment of β-Hemoglobinopathies

Jean-Yves Metais, PhD, Phillip A Doerfler, PhD, Thiyagaraj Mayuranathan, Daniel E. Bauer, MD PhD, Stephanie Fowler, Matthew Hsieh, MD, Varun Katta, Sagar Keriwala, Cicera Lazzarotto, Kevin Luk, Michael Neel, Scott Perry, Shaina Porter, Byoung Y. Ryu, PhD, Akshay Sharma, MBBS, Devlin Shea, Samuel Peters, John F. Tisdale, MD, Scot A. Wolfe, PhD, Kaitly Jensen Woodard, BS, Naoya Uchida, MD, Yuxuan Wu, PhD, Yu Yao, MD, Jing Zeng, MD, Shondra M. Pruett-Miller, PhD, Shengdar Q. Tsai, PhD, Mitchell J. Weiss, MD, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2066.
https://doi.org/10.1182/blood-2019-131318
Published: 2019
Journal Articles

Safe and Efficient Peripheral Blood Stem Cell Collection in Patients with Sickle Cell Disease Using Plerixafor

Alexis Leonard, MD, Naoya Uchida, MD, David F. Stroncek, MD, Sandhya R. Panch, MDMPH, Kamille West, Eoghan George Molloy, MD, Tom Hughes, Pharm.D., Sara Hauffe, RN, Tiffani Taylor, Courtney D. Fitzhugh, MD, Jane S. Hankins, MD MS, Megan Wilson, MSW, CCRP, Akshay Sharma, MBBS, Shengdar Q. Tsai, PhD, Mitchell J. Weiss, MD, PhD, Matthew Hsieh, MD, John F. Tisdale, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1964.
https://doi.org/10.1182/blood-2019-126091
Published: 2019