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Journal Articles

Introduction to a review series on gene therapy and gene editing for sickle cell disease and hemophilia

Catherine M. Bollard

Blood (2021) 138 (11): 913.

DOI: https://doi.org/10.1182/blood.2021012891

Published: 2021

Journal Articles

Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells

Blood (2021) 137 (19): 2598–2608.

DOI: https://doi.org/10.1182/blood.2020008503

Published: 2021

Includes: Supplemental data

Journal Articles

Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia

Angela R. Smith, MD MS, Gary J. Schiller, MD, Gregory M Vercellotti, MD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Erica B. Esrick, MD, David A. Williams, MD, Weston P. Miller, MD, Adrian Woolfson, MD PhD, Mark C. Walters, MD

Blood (2019) 134 (Supplement_1): 3544.

DOI: https://doi.org/10.1182/blood-2019-125743

Published: 2019

Journal Articles

Gene Editing and mRNA-Based Therapy: Two Complementary Therapeutic Approaches for the Treatment of Patients with Xmen Disease

Julie Brault, PhD PharmD, Ezekiel Bello, Ronald J. Meis, PhD, Taylor Liu, Sherry M. Koontz, BS, Kennichi Dowdell, PhD, Linhong Li, PhD, Cornell Allen, MS, Narda Theobald, BS, Janet Lee, BS, Aaron B. Clark, BS, Juan C. Ravell, MD, Michael Lenardo, MD, Gary A. Dahl, PhD, Harry L. Malech, MD, Suk See De Ravin, MD, PhD

Blood (2019) 134 (Supplement_1): 4637.

DOI: https://doi.org/10.1182/blood-2019-129062

Published: 2019

Journal Articles

NHEJ-Mediated Gene Editing, a Versatile Approach to Correct a Variety of Fanconi Anemia Genes in HSCs

Francisco J Roman-Rodriguez, PhD, Laura Ugalde, Lara A. Alvarez, Begoña Díez-Cabezas, PhD, Jordi Surrallés, PhD, Maria Jose Ramirez, PhD, Massimo Bogliolo, PhD, Helmut Hanenberg, MD PhD, Julian Sevilla, MD PhD, Sandra Rodriguez-Perales, PhD, Raul Torres-Ruiz, PhD, Juan A. Bueren, PhD, Paula Rio, PhD

Blood (2019) 134 (Supplement_1): 4639.

DOI: https://doi.org/10.1182/blood-2019-129946

Published: 2019

Journal Articles

Identification of Optimized Peptide Nucleic Acid (PNA) Designs for Nuclease-Free Gene Editing in Sickle Cell Disease Hematopoietic Stem and Progenitor Cells (HSPCs)

Allen Ebens, PhD, Andrew Curd, B.Sc., Derek Sim, PhD, Elias Quijano, B.Sc., Florence Tam, M.Sc., James Coull, PhD,, Lily Huang, M.Sc., Mark Hayes, PhD, Marshall Fordyce, MD, Robert Pearson, B.Sc., Susan Whoriskey, PhD, Tumul Srivastava, PhD, Xiaojun Li, MD, Zhen Zhang, PhD

Blood (2019) 134 (Supplement_1): 4645.

DOI: https://doi.org/10.1182/blood-2019-127538

Published: 2019

Journal Articles

Venetoclax Resistance in Mantle Cell Lymphoma Is Mediated By BCL-XL and Can be Circumvent By Inhibiting the BH4 Domain of BCL-2

Daniela Steinbrecher, PhD, Felix Seyfried, MD, Eugen Tausch, Johannes Bloehdorn, MD, Billy Michael Chelliah Jebaraj, PhD, Klaus-Michael Debatin, MD, Lüder Hinrich Meyer, MD, Hartmut Döhner, MD, Stephan Stilgenbauer, MD, Christof Schneider, MD

Blood (2019) 134 (Supplement_1): 1507.

DOI: https://doi.org/10.1182/blood-2019-127931

Published: 2019

Journal Articles

Heterozygous Mutations of Clpb As a Newly Identified and Frequent Cause of Severe Congenital Neutropenia

Julia T Warren, MD PhD, Peeradol Wattanasirakul, David H Spencer, MD, Adam Locke, PhD, Vahagn Makaryan, MD, Audrey Anna Bolyard, RN, BS, Karl Welte, Julia Skokowa, MD PhD, David C. Dale, MD, Daniel C. Link, MD

Blood (2019) 134 (Supplement_1): 433.

DOI: https://doi.org/10.1182/blood-2019-129635

Published: 2019

Journal Articles

Disease Modeling of Severe Congenital Neutropenia Using CRISPR/Cas9 Gene Correction or Knockout of ELANE in Patients Derived Induced Pluripotent Stem Cells

Masoud Nasri, MSc, Benjamin Dannenmann, Perihan Mir, Malte U Ritter, Diana Amend, Yun Xu, Maksim Klimiankou, Cornelia Zeidler, Karl Welte, Julia Skokowa, MD PhD

Blood (2019) 134 (Supplement_1): 434.

DOI: https://doi.org/10.1182/blood-2019-129991

Published: 2019

Journal Articles

CRISPR/Cas9 Mediated ELANE Knock-out Restores Survival and Granulocytic Differentiation of HL60 Cells Expressing Mutant Neutrophil Elastase: Is Neutrophil Elastase a Dispensible Granulocyte Protease?

Vahagn Makaryan, MD, Isabella N. Archibald, Merideth L. Kelley, MS, Breanna Fletcher, BSc, David C. Dale, MD

Blood (2019) 134 (Supplement_1): 435.

DOI: https://doi.org/10.1182/blood-2019-124988

Published: 2019

Journal Articles

Inherited GATA3 Variants Associated with Positive Minimal Residual Disease in Childhood B-ALL Via Autophagy-Induced Asparaginase Resistance

Chunjie Li, PhD, Jiabi Qian, M.Sc, Chuang Jiang, PhD, Ziping LI, B.S, Hui Zhang, MD PhD

Blood (2019) 134 (Supplement_1): 654.

DOI: https://doi.org/10.1182/blood-2019-123575

Published: 2019

Journal Articles

Zinc Finger Nuclease-Mediated Disruption of the BCL11A Erythroid Enhancer Results in Enriched Biallelic Editing, Increased Fetal Hemoglobin, and Reduced Sickling in Erythroid Cells Derived from Sickle Cell Disease Patients

Samuel Lessard, PhD, Pauline Rimmele, PhD, Hui Ling, PhD, Kevin Moran, MSc, Benjamin Vieira, BS, Yi-Dong Lin, PhD, Vu Hong, PhD, Andreas Reik, PhD, Denny Dang, Naoya Uchida, MD, John F. Tisdale, MD, Pablo Rendo, MD, Ahmed Daak, PhD, Alexandra Hicks, PhD

Blood (2019) 134 (Supplement_1): 974.

DOI: https://doi.org/10.1182/blood-2019-126048

Published: 2019

Journal Articles

Chromosomal Loop Editing Reveals New Epigenomic Landscape in the β-Globin Locus with Implications for Hemoglobinopathy Therapies

Pamela Himadewi, BSc, Xiaotian Zhang, PhDBS, Haley Gore, MS, Xue Qing David Wang, PhD

Blood (2019) 134 (Supplement_1): 813.

DOI: https://doi.org/10.1182/blood-2019-125020

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.

DOI: https://doi.org/10.1182/blood-2019-131073

Published: 2019

Journal Articles

First-in-Human Assessment of Feasibility and Safety of Multiplexed Genetic Engineering of Autologous T Cells Expressing NY-ESO -1 TCR and CRISPR/Cas9 Gene Edited to Eliminate Endogenous TCR and PD-1 (NYCE T cells) in Advanced Multiple Myeloma (MM) and Sarcoma

Blood (2019) 134 (Supplement_1): 49.

DOI: https://doi.org/10.1182/blood-2019-122374

Published: 2019

Journal Articles

Characterization of a Genetically Engineered HUDEP2 Cell Line Harboring a Sickle Cell Disease Mutation As a Potential Research Tool for Preclinical Sickle Cell Disease Drug Discovery

Dipti Gupta, Sarah Sturtevant, BSc, Benjamin Vieira, BS, Yukio Nakamura, MD PhD, Sriram Krishnamoorthy, PhD, Melanie Demers, PhD

Blood (2019) 134 (Supplement_1): 3559.

DOI: https://doi.org/10.1182/blood-2019-127330

Published: 2019

Journal Articles

Next Generation CRISPR Gene-Edited and Off-the-Shelf Virus-Specific T-Cells for the Immunocompromised Patient

Blood (2019) 134 (Supplement_1): 1944.

DOI: https://doi.org/10.1182/blood-2019-125276

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.

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

Published: 2019

Journal Articles

Enabling Gene-Edited, Regulatory-like, T Cells (edTreg) for Treatment of IPEX and Other Autoimmune Disorders

Yuchi Honaker, PhD, Karen Sommer, PhD, Noelle Dahl, Yufei Xiang, MD, PhD, Christina Lopez, PhD, Samuel Scharffenberger, Michelle Christian, Troy R. Torgerson, MD PhD, Andrew M. Scharenberg, MD, David J. Rawlings, MD

Blood (2019) 134 (Supplement_1): 2071.

DOI: https://doi.org/10.1182/blood-2019-131946

Published: 2019

Journal Articles

Infection Is a Driver of Dnmt3a-Mutant Clonal Hematopoiesis

Daniel Hormaechea Agulla, PhD, Katie A Matatall, PhD, Duy Le, PhDcandidate, Bailee Nicole Kain, PhDcandidate, Roman Jaksik, PhD, Marek Kimmel, PhD DSc, Grant Challen, PhD, Katherine Y. King, MD PhD

Blood (2019) 134 (Supplement_1): 817.

DOI: https://doi.org/10.1182/blood-2019-126831

Published: 2019

NARROW

Introduction to a review series on gene therapy and gene editing for sickle cell disease and hemophilia

Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells

Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia

Gene Editing and mRNA-Based Therapy: Two Complementary Therapeutic Approaches for the Treatment of Patients with Xmen Disease

NHEJ-Mediated Gene Editing, a Versatile Approach to Correct a Variety of Fanconi Anemia Genes in HSCs

Identification of Optimized Peptide Nucleic Acid (PNA) Designs for Nuclease-Free Gene Editing in Sickle Cell Disease Hematopoietic Stem and Progenitor Cells (HSPCs)

Venetoclax Resistance in Mantle Cell Lymphoma Is Mediated By BCL-XL and Can be Circumvent By Inhibiting the BH4 Domain of BCL-2

Heterozygous Mutations of Clpb As a Newly Identified and Frequent Cause of Severe Congenital Neutropenia

Disease Modeling of Severe Congenital Neutropenia Using CRISPR/Cas9 Gene Correction or Knockout of ELANE in Patients Derived Induced Pluripotent Stem Cells

CRISPR/Cas9 Mediated ELANE Knock-out Restores Survival and Granulocytic Differentiation of HL60 Cells Expressing Mutant Neutrophil Elastase: Is Neutrophil Elastase a Dispensible Granulocyte Protease?

Inherited GATA3 Variants Associated with Positive Minimal Residual Disease in Childhood B-ALL Via Autophagy-Induced Asparaginase Resistance

Zinc Finger Nuclease-Mediated Disruption of the BCL11A Erythroid Enhancer Results in Enriched Biallelic Editing, Increased Fetal Hemoglobin, and Reduced Sickling in Erythroid Cells Derived from Sickle Cell Disease Patients

Chromosomal Loop Editing Reveals New Epigenomic Landscape in the β-Globin Locus with Implications for Hemoglobinopathy Therapies

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

First-in-Human Assessment of Feasibility and Safety of Multiplexed Genetic Engineering of Autologous T Cells Expressing NY-ESO -1 TCR and CRISPR/Cas9 Gene Edited to Eliminate Endogenous TCR and PD-1 (NYCE T cells) in Advanced Multiple Myeloma (MM) and Sarcoma

Characterization of a Genetically Engineered HUDEP2 Cell Line Harboring a Sickle Cell Disease Mutation As a Potential Research Tool for Preclinical Sickle Cell Disease Drug Discovery

Next Generation CRISPR Gene-Edited and Off-the-Shelf Virus-Specific T-Cells for the Immunocompromised Patient

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

Enabling Gene-Edited, Regulatory-like, T Cells (edTreg) for Treatment of IPEX and Other Autoimmune Disorders

Infection Is a Driver of Dnmt3a-Mutant Clonal Hematopoiesis

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American Society of Hematology

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American Society of Hematology

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