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Journal Articles

Adenine Base Editing Improves Erythropoiesis in Diamond-Blackfan Anemia Syndrome Patient-Derived Induced Pluripotent Stem Cells

Shruthi Suryaprakash, Lei Han, Garret Manquen, Varun Katta, Damian Krzyzanowski, Jayaram Prasad, Nikitha Nimmagada, Kalin Mayberry, Nana Liu, Yan Ju, Yu Yao, Kelsey Ray, Marcin Wlodarski, Shengdar Q Tsai, Jonathan S Yen, Mitchell J Weiss, Senthil Velan Bhoopalan
Journal: Blood
Blood (2024) 144 (Supplement 1): 4088.
https://doi.org/10.1182/blood-2024-209312
Published: 2024
Journal Articles

SAGES1: Clinical Translation of CRISPR Genome Editing Strategy to Induce Fetal Hemoglobin to Treat Sickle Cell Disease

Rachel M Levine, Erin A Dempsey, Nikitha Nimmagadda, Yoonjeong Jang, Kalin Mayberry, Jingjing Zhang, David Cullins, Catherine Willis, Alicia Powers, Young-In Kim-Hoehammer, Jie Lin, Fei Zhang, Neha Das Gupta, Will Lewis, Varun Katta, Yichao Li, Timothy Lockey, Yong Cheng, Patti Lehigh, Frank Fazio, Alexis Kristine Leonard, Akshay Sharma, Sheng Zhou, Shengdar Q Tsai, Mitchell J Weiss, Jonathan S Yen
Journal: Blood
Blood (2024) 144 (Supplement 1): 146.
https://doi.org/10.1182/blood-2024-211182
Published: 2024
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

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