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Journal Articles

Efficacy and Safety of Brl-101, CRISPR-Cas9-Mediated Gene Editing of the BCL11A Enhancer in Transfusion-Dependent β-Thalassemia

Biao Zheng, Rongrong Liu, Xinhua Zhang, Bin Fu, Yajing Xu, Jun Shi, Xiaoqin Feng, Li Wang, Chris Wang, Rongmei Liang, Lingzhi Tan, Fei Wang, Dali Li, Yuxuan Wu, Mingyao Liu, Yongrong Lai
Journal: Blood
Blood (2023) 142 (Supplement 1): 4995.
https://doi.org/10.1182/blood-2023-186031
Published: 2023
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

Therapeutic Base Editing of Human Hematopoietic Stem Cells

Jing Zeng, MD, Yuxuan Wu, PhD, Chunyan Ren, PhD, Jasmine Bonanno, Anne H. Shen, Devlin Shea, Jason M Gehrke, PhD, John P Manis, MD, J. Keith Gehrke, Daniel E. Bauer, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 612.
https://doi.org/10.1182/blood-2019-129173
Published: 2019
Journal Articles

Gene Editing ELANE in Human Hematopoietic Stem and Progenitor Cells Reveals Disease Mechanisms and Therapeutic Strategies for Severe Congenital Neutropenia

Shuquan Rao, PhD, Josias Brito-Frazao, Anna V. Serbin, Qiuming Yao, PhD, Kevin Luk, Yuxuan Wu, PhD, Jing Zeng, MD, Chunyan Ren, PhD, Ruth Watkinson, Myriam Armant, PhD, Luca Pinello, PhD, Akiko Shimamura, MD PhD, Benhur Lee, Scot A. Wolfe, PhD, Peter E. Newburger, MD, Daniel E. Bauer, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3.
https://doi.org/10.1182/blood-2019-131073
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

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

Editing aberrant splice sites efficiently restores β-globin expression in β-thalassemia

Brief Report
Shuqian Xu, Kevin Luk, Qiuming Yao, Anne H. Shen, Jing Zeng, Yuxuan Wu, Hong-Yuan Luo, Christian Brendel, Luca Pinello, David H. K. Chui, Scot A. Wolfe, Daniel E. Bauer
Journal: Blood
Blood (2019) 133 (21): 2255–2262.
https://doi.org/10.1182/blood-2019-01-895094
Published: 2019
Includes: Supplemental data
Journal Articles

Comprehensive Integrated Genomic Perturbations Reveal Molecular Mechanisms of Red Blood Cell Trait Associations

Yuxuan Wu, PhD, Mitchel A. Cole, BS, Abdou Mousas, Jing Zeng, MD, Qiuming Yao, PhD, Divya Vinjamur, PhD, Ryo Kurita, PhD, Yukio Nakamura, MD PhD, Luca Pinello, PhD, Guillame Lettre, PhD, Daniel E. Bauer, MD PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 532.
https://doi.org/10.1182/blood-2018-99-120229
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

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