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Journal Articles

Epitope Editing Combined with Extended Schedule Anti-KIT Antibody Treatment Allows Immune-Based In Vivo Selection of Multiplex Genome-Engineered Cells

Gabriele Casirati, Adele Mucci, Marta Freschi, Andrea Cosentino, Mohammed S Mahmoud, Jing Zeng, Nola Neri, Christian Brendel, Daniel Bauer, Pietro Genovese
Journal: Blood
Blood (2024) 144 (Supplement 1): 4767.
https://doi.org/10.1182/blood-2024-202480
Published: 2024
Journal Articles

Epitope Edited Hematopoietic Stem Cells to Enable Synergistic Immunotherapy Combinations for Acute Myeloid Leukemia

Gabriele Casirati, Andrea Cosentino, Adele Mucci, Mohammed S Mahmoud, Iratxe Ugarte Zabala, Jing Zeng, Scott Ficarro, Denise Klatt, Christian Brendel, Alessandro Rambaldi, Jerome Ritz, Jarrod Marto, Danilo Pellin, Daniel E. Bauer, Scott A Armstrong, Pietro Genovese
Journal: Blood
Blood (2023) 142 (Supplement 1): 7130.
https://doi.org/10.1182/blood-2023-189980
Published: 2023
Journal Articles

UM171 Enhances Fitness and Engraftment of Gene Modified Hematopoietic Stem Cells from Sickle Cells Disease Patients

Boya Liu, Denise Klatt, Yu Zhou, John Manis, Guy Sauvageau, Christian Brendel, David A. Williams
Journal: Blood
Blood (2023) 142 (Supplement 1): 18.
https://doi.org/10.1182/blood-2023-182099
Published: 2023
Journal Articles

Epitope Edited Hematopoietic Stem Cells Allow Immune-Based In Vivo Selection of Genome-Engineered Cells

Gabriele Casirati, Andrea Cosentino, Adele Mucci, Mohammed S Mahmoud, Marta Freschi, Jing Zeng, Christian Brendel, Daniel E. Bauer, Pietro Genovese
Journal: Blood
Blood (2023) 142 (Supplement 1): 7127.
https://doi.org/10.1182/blood-2023-177772
Published: 2023
Journal Articles

Combining the Anti-Sickling βAS3-Globin Gene with BCL11A and ZNF410 Double Shmirs for the Treatment of Sickle Cell Disease

Boya Liu, Kevyn Hart, Christian Brendel, Donald B. Kohn, David A. Williams
Journal: Blood
Blood (2022) 140 (Supplement 1): 7786–7787.
https://doi.org/10.1182/blood-2022-163469
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

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

Induction of Fetal Hemoglobin and Reduction of Clinical Manifestations in Patients with Severe Sickle Cell Disease Treated with Shmir-Based Lentiviral Gene Therapy for Post-Transcriptional Gene Editing of BCL11A: Updated Results from Pilot and Feasibility Trial

Erica B. Esrick, Amy Federico, Daniela Abriss, Myriam Armant, Kari Boardman, Christian Brendel, Marioara-Felicia Ciuculescu, Heather Daley, Colleen Dansereau, Augustine Fernandes, Matthew M. Heeney, David G. Justus, Pei-Chi Kao, Annette S. Kim, Donald B. Kohn, Leslie E. Lehmann, Donghui Liu, Wendy B. London, John P Manis, Theodore B. Moore, Emily Morris, Danilo Pellin, Ellen Proeung, Shanna Richard, Gavin D. Roach, Kit L. Shaw, Dayna Terrazas, Shanna L White, David A. Williams
Journal: Blood
Blood (2022) 140 (Supplement 1): 10665–10667.
https://doi.org/10.1182/blood-2022-169089
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

Epitope Engineered Hematopoietic Stem and Progenitor Cells to Enable CAR-T Cell Immunotherapy for Acute Myeloid Leukemia

Gabriele Casirati, Andrea Cosentino, Adele Mucci, Iraxte Ugarte Zabala, Mohammed S. Mahmoud, Christian Brendel, Alessandro Rambaldi, Jerome Ritz, Scott A. Armstrong, Pietro Genovese
Journal: Blood
Blood (2022) 140 (Supplement 1): 303–304.
https://doi.org/10.1182/blood-2022-167285
Published: 2022
Journal Articles

An Inducible Leukemia-Associated Transcription Factor Facilitates Large-Scale Ex Vivo Generation of Functional Human Macrophages

Roland Windisch, Sarah Soliman, Adrian Hoffmann, Linping Chen-Wichmann, Sebastian Lutz, Christian Kellner, Enric Redondo Monte, Sebastian Vosberg, Luise Hartmann, Stephanie Schneider, Fabian Beier, Carolin Dorothea Strobl, Oliver Weigert, Matthias Peipp, Michael Schündeln, Jürgen Bernhagen, Andreas Humpe, Christian Brendel, Hannes Klump, Philipp A. Greif, Christian Wichmann
Journal: Blood
Blood (2021) 138 (Supplement 1): 2805.
https://doi.org/10.1182/blood-2021-148336
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

Effects of BCL11A Shmir-Induced Post-Transcriptional Silencing on Hemoglobin Polymer Inhibition in Single Red Blood Cells at Physiologic Oxygen Tension

Daniel C. De Souza, Erica B. Esrick, Nicolas Hebert, Giuseppe Di Caprio, Marioara Ciuculescu, Emily Morris, Myriam Armant, Bronner P. Gonçalves, Ethan Schonbrun, Christian Brendel, David K. Wood, Pablo Bartolucci, David A. Williams, John M. Higgins
Journal: Blood
Blood (2021) 138 (Supplement 1): 964.
https://doi.org/10.1182/blood-2021-147314
Published: 2021
Journal Articles

Effects of BCL11A Shmir-Induced Post-Transcriptional Silencing on Distributions of HbF in Single-RBCs and Reticulocytes

Nicolas Hebert, Erica B. Esrick, Myriam Armant, Christian Brendel, Marioara Felicia Ciuculescu, Etienne Audureau, John M. Higgins, David A. Williams, Pablo Bartolucci
Journal: Blood
Blood (2021) 138 (Supplement 1): 967.
https://doi.org/10.1182/blood-2021-150186
Published: 2021
Journal Articles

Distinct Genetic Pathways Define Leukemia Predisposition Versus Adaptive Clonal Hematopoiesis in Shwachman-Diamond Syndrome

Alyssa L. Kennedy, MDPhD, Kasiani C. Myers, MD, James Bowman, PhD, Christopher J. Gibson, MD, Gwen M. Muscato, Robert Klein, Kaitlyn Ballotti, Nicholas Camarda, Elissa M. Furutani, MD, Chad E. Harris, MS, Shanshan Liu, PhD, Ashley Galvin, Maggie Malsch, MSN, RN, David C. Dale, MD, John M Gansner, MD PhD, Taizo A. Nakano, MD, Alison A. Bertuch, MD, Adrianna Vlachos, MD, Jeff H. Lipton, MD PhD, Paul Castillo, MD, James Connelly, John Edwards, MD, Nobuko Hijiya, MD, Richard Ho, MD, Inga Hofmann, MD, James N Huang, MD, Sioban B. Keel, MD, Adam J. Lamble, MD, Bonnie W Lau, MD PhD, Kelly J. Walkovich, MD, Maxim Norkin, MD, Wendy Stock, MD, Steffen Boettcher, Christian Brendel, PhD, Elliot Stieglitz, MD, Mark D. Fleming, MD DPhil, Stella M. Davies, PhD MBBS, MRCP, Edie A. Weller, Chris Bahl, PhD, Scott L. Carter, PhD, Akiko Shimamura, MD PhD, R. Coleman Lindsley, MD PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 35–36.
https://doi.org/10.1182/blood-2020-137125
Published: 2020
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

Deletion of Murine Rhoh induces More Aggressive Diffuse Large B Cell Lymphoma (DLBCL) Via Interaction with Kaiso and Regulation of BCL-6 Expression

Hiroto Horiguchi, MDPhD, Marioara Felicia Ciuculescu, MD, Anja Troeger, MD, Haiming Xu, PhD, Christian Brendel, PhD, David A. Williams, MD
Journal: Blood
Blood (2018) 132 (Supplement 1): 1574.
https://doi.org/10.1182/blood-2018-99-115238
Published: 2018
Journal Articles

Interleukin-10 Signaling in Hematopoietic Stem and Progenitor Cells Maintains Stem Cell Function and Regulates Inflammation-Induced Myeloid Cell Output

Amanda McCabe, PhD, Christian Brendel, PhD, Sandra Frei, PhD, Denise Klatt, MSc, Martin Bentler, Scott Snapper, MD PhD, David A. Williams, MD
Journal: Blood
Blood (2018) 132 (Supplement 1): 2407.
https://doi.org/10.1182/blood-2018-99-116168
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
Journal Articles

Flipping the Switch: Initial Results of Genetic Targeting of the Fetal to Adult Globin Switch in Sickle Cell Patients

Erica B. Esrick, MD, Christian Brendel, PhD, John P Manis, MD, Myriam A Armant, PhD, Helene Negre, PhD, Colleen Dansereau, MSN, RN, CPN, Marioara Felicia Ciuculescu, MD, Stephanie Patriarca, Brenda Mackinnon, BSN, RN, Heather Daley, BS, Shanna Richard, Daniela Abriss, Renee Maxwell, Stephen Braunewell, BS, Lauryn Christiansen, BS, Sarah Nikiforow, MD PhD, Jerome Ritz, MD PhD, Maureen Achebe, MD, Olivier Negre, PhD, Leslie E. Lehmann, MD, Matthew M Heeney, MD, Alessandra Biffi, MD PhD, David A. Williams, MD
Journal: Blood
Blood (2018) 132 (Supplement 1): 1023.
https://doi.org/10.1182/blood-2018-99-116733
Published: 2018
Journal Articles

Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown, β-Globin Induction and Simultaneous Repression of β-Globin for the Potential Treatment of Sickle Cell Disease

Olivier Negre, PhD, Christian Brendel, PhD, Geoffrey B. Parsons, Min Luo, PhD, Myriam A Armant, PhD, Swaroopa Guda, PhD, Lauryn Christiansen, Gretchen Lewis, BMBS, Gabor Veres, PhD, Melissa Bonner, PhD, David A. Williams, MD
Journal: Blood
Blood (2017) 130 (Supplement 1): 3557.
https://doi.org/10.1182/blood.V130.Suppl_1.3557.3557
Published: 2017