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Scot A. Wolfe
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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

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

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

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

Germline Runx1 Mutations Induce Inflammation in Hematopoietic Stem and Progenitor Cells and Predispose to Hematologic Malignancies

Mohd Hafiz Ahmad, Mahesh Hegde, Waihay J. Wong, Andrew Dunbar, Anneliese Carrascoso, Lisa J Garrett, Nayara K Ferreira, Haibo Liu, Rui Li, Benjamin L. Ebert, Ross L. Levine, Julie Zhu, Scot A. Wolfe, Paul P Lui, Lucio H. Castilla
Journal: Blood
Blood (2021) 138 (Supplement 1): 2201.
https://doi.org/10.1182/blood-2021-153433
Published: 2021
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

The CBFβ-SMMHC/NRP1 Axis Regulates FLT3 and TGF-Beta Pathways in Inv(16) Acute Myeloid Leukemia

Mahesh Hegde, Mohd Hafiz Ahmad, Rui Li, Anneliese Carrascoso, Roger Mulet-Lazaro, Ruud Delwel, John H. Bushweller, Monica L. Guzman, Scot A. Wolfe, Julie Zhu, Lucio H. Castilla
Journal: Blood
Blood (2021) 138 (Supplement 1): 3314.
https://doi.org/10.1182/blood-2021-153275
Published: 2021
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

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
Journal Articles

CBFβ-SMMHC Inhibition Disrupts Enhancer Chromatin Dynamics and Represses MYC Transcriptional Program in Inv(16) Leukemia

John Anto Pulikkan, PhD, Mahesh Hegde, Houda Belaghzal, Anuradha Illendula, Jun Yu, Hafiz Ahmed, Kelsey O'Hagen, Jianhong Ou, Carsten Müller-Tidow, Scot A. Wolfe, Julie Zhu, Job Dekker, John H. Bushweller, Lucio H. Castilla
Journal: Blood
Blood (2017) 130 (Supplement 1): 784.
https://doi.org/10.1182/blood.V130.Suppl_1.784.784
Published: 2017