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Journal Articles

IGF2BP1 Orchestrates Post-Transcriptional Control of Globin Switching through Direct Binding to Globins and Their Regulators

Steven Coyne, Ting Wu, Divya Vinjamur, Mir Hossain, Daniel E. Bauer

Blood (2024) 144 (Supplement 1): 414.

https://doi.org/10.1182/blood-2024-210473

Published: 2024

Journal Articles

A Novel Small Molecule Drug-Based Approach for Treating Beta-Globinopathies

Jun Liu, Ziyang Shen, Yanhua Dong, Na Yu, Li Chai, Daniel G. Tenen, Daniel E. Bauer, Adelaide Herman, Braden Pate, Kim Vanuytsel

Blood (2024) 144 (Supplement 1): 172.

https://doi.org/10.1182/blood-2024-211753

Published: 2024

Journal Articles

Genetic medicine gestating for α-thalassemia

Daniel E. Bauer

Blood (2024) 144 (15): 1554–1556.

https://doi.org/10.1182/blood.2024025938

Published: 2024

Journal Articles

Modulating Nucleotide Metabolism Enhances Prime Editing in Hematopoietic Stem and Progenitor Cells

Sébastien Levesque, Daniel E. Bauer

Blood (2023) 142 (Supplement 1): 7124.

https://doi.org/10.1182/blood-2023-190750

Published: 2023

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

Blood (2023) 142 (Supplement 1): 7130.

https://doi.org/10.1182/blood-2023-189980

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

Blood (2023) 142 (Supplement 1): 7127.

https://doi.org/10.1182/blood-2023-177772

Published: 2023

Journal Articles

A viable alternative for editor delivery

Feyisayo Eweje, Daniel E. Bauer

Blood (2023) 142 (9): 755–756.

https://doi.org/10.1182/blood.2023021309

Published: 2023

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

Blood (2022) 140 (Supplement 1): 10671–10672.

https://doi.org/10.1182/blood-2022-169900

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

Blood (2022) 140 (Supplement 1): 2944–2945.

https://doi.org/10.1182/blood-2022-169379

Published: 2022

Journal Articles

Clonal hematopoiesis in sickle cell disease

Thomas Pincez, Simon S. K. Lee, Yann Ilboudo, Michael Preuss, Anne-Laure Pham Hung d’Alexandry d’Orengiani, Pablo Bartolucci, Frédéric Galactéros, Philippe Joly, Daniel E. Bauer, Ruth J. F. Loos, R. Coleman Lindsley, Guillaume Lettre

Blood (2021) 138 (21): 2148–2152.

https://doi.org/10.1182/blood.2021011121

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

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

Blood (2021) 138 (Supplement 1): 1852.

https://doi.org/10.1182/blood-2021-152634

Published: 2021

Journal Articles

ZNF410 Represses Fetal Globin By Devoted Control of CHD4/NuRD

Divya S Vinjamur, PhD, Qiuming Yao, PhD, Mitchel A. Cole, BS, Connor McGuckin, Chunyan Ren, PhD, Jing Zeng, MD, Mir Hossain, PhD, Luca Pinello, PhD, Daniel E Bauer, MD PhD

Blood (2020) 136 (Supplement 1): 1.

https://doi.org/10.1182/blood-2020-142134

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

Blood (2019) 134 (Supplement_1): 4632.

https://doi.org/10.1182/blood-2019-129394

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

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

Blood (2019) 134 (Supplement_1): 2066.

https://doi.org/10.1182/blood-2019-131318

Published: 2019

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

Blood (2019) 134 (Supplement_1): 612.

https://doi.org/10.1182/blood-2019-129173

Published: 2019

Journal Articles

CRISPR-Cas9 Screen Identifies XPO7 As a Potential Therapeutic Target for TP53 -Mutated AML

Yuichiro Semba, MD PhD, Takuji Yamauchi, MD PhD, Fumihiko Nakao, MD, Jumpei Nogami, PhD, Matthew C. Canver, MD PhD, Luca Pinello, PhD, Daniel E. Bauer, MD PhD, Koichi Akashi, MD PhD, Takahiro Maeda, MD PhD

Blood (2019) 134 (Supplement_1): 3784.

https://doi.org/10.1182/blood-2019-129623

Published: 2019

Journal Articles

Paics, a De Novo Purine Synthetic Enzyme, Is a Novel Target for AML Therapy

Takuji Yamauchi, MD PhD, Kohta Miyawaki, MD, Yuichiro Semba, MD PhD, Fumihiko Nakao, MD, Takeshi Sugio, MD PhD, Kensuke Sasaki, MD, Jumpei Nogami, PhD, Matthew C. Canver, MD PhD, Simon Osborne, PhD, Luca Pinello, PhD, Deborah Taylor, PhD, Daniel E. Bauer, MD PhD, Koichi Akashi, MD PhD, Takahiro Maeda, MD PhD

Blood (2019) 134 (Supplement_1): 1390.

https://doi.org/10.1182/blood-2019-127047

Published: 2019

Journal Articles

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

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

Blood (2019) 133 (21): 2255–2262.

https://doi.org/10.1182/blood-2019-01-895094

Published: 2019

Includes: Supplemental data

NARROW

IGF2BP1 Orchestrates Post-Transcriptional Control of Globin Switching through Direct Binding to Globins and Their Regulators

A Novel Small Molecule Drug-Based Approach for Treating Beta-Globinopathies

Genetic medicine gestating for α-thalassemia

Modulating Nucleotide Metabolism Enhances Prime Editing in Hematopoietic Stem and Progenitor Cells

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

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

A viable alternative for editor delivery

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

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

Clonal hematopoiesis in sickle cell disease

Human Genetic Diversity Alters Therapeutic Gene Editing Off-Target Outcomes

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

ZNF410 Represses Fetal Globin By Devoted Control of CHD4/NuRD

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

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

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

Therapeutic Base Editing of Human Hematopoietic Stem Cells

CRISPR-Cas9 Screen Identifies XPO7 As a Potential Therapeutic Target for TP53 -Mutated AML

Paics, a De Novo Purine Synthetic Enzyme, Is a Novel Target for AML Therapy

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

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American Society of Hematology

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ASH Publications

American Society of Hematology

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