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Journal Articles

ILF2 Antisense Oligonucleotide Therapy and a CRISPR/Cas9-Based Screening for DNA Repair Effectors Identify Synthetic Lethal Approaches Enhancing Myeloma Cells Sensitivity to DNA Damage

Natthakan Thongon, PhD, Christopher Jackson, BS, Pamela Lockyer, MS, Jintan Liu, Andrea Santoni, PhD, Ashley Rose, BS, Guillermo Garcia-Manero, MD, Simona Colla, PhD

Blood (2019) 134 (Supplement_1): 685.

https://doi.org/10.1182/blood-2019-129286

Published: 2019

Journal Articles

Loss of METTL3 Mediated m6A RNA Modification Results in Double-Stranded RNA Induced Innate Immune Response and Hematopoietic Failure

Blood (2019) 134 (Supplement_1): 450.

https://doi.org/10.1182/blood-2019-130442

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

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

Molecular Characterization and Novel Therapeutic Strategy for X-Linked Sideroblastic Anemia Associated with ALAS2 Missense Variants

Koya Ono, MD, Tohru Fujiwara, MD PhD, Kei Saito, MD PhD, Chie Suzuki, Noriyuki Takahashi, Koichi Onodera, MD PhD, Satoshi Ichikawa, MD PhD, Noriko Fukuhara, MD PhD, Yasushi Onishi, MD PhD, Hisayuki Yokoyama, MD PhD, Yukio Nakamura, MD PhD, Hideo Harigae, MD PhD

Blood (2019) 134 (Supplement_1): 3530.

https://doi.org/10.1182/blood-2019-124810

Published: 2019

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.

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

Published: 2019

Journal Articles

Disease Modeling and Phenotype Rescue Using Inducible Pluripotent Stem Cells from Patients with Diamond-Blackfan Anemia

Senthil Velan Bhoopalan, MD PhD, Min-Joon Han, PhD, Steven Ellis, PhD, Harry Lesmana, MD, Jeremie H. Estepp, MD, Yoonjeong Jang, DVM,PhD, Mitchell J. Weiss, MD PhD, Marcin W. Wlodarski, MD PhD

Blood (2019) 134 (Supplement_1): 2496.

https://doi.org/10.1182/blood-2019-131354

Published: 2019

Journal Articles

CRISPR/Cas9-Targeted De Novo DNA Methylation Is Maintained and Impacts the Colony Forming Potential of Human Hematopoietic CD34 + Cells

Emily A Saunderson, PhDBSc, Kevin Rouault-Pierre, PhDBSc, John G. Gribben, MD DSc, Gabriella Ficz, PhDBSc

Blood (2019) 134 (Supplement_1): 2517.

https://doi.org/10.1182/blood-2019-130267

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.

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

Published: 2019

Journal Articles

Polymorphisms in Inflammatory Genes Modulate the Occurrence of Clinical Complications in Patients with Sickle Cell Disease

Blood (2019) 134 (Supplement_1): 4825.

https://doi.org/10.1182/blood-2019-124218

Published: 2019

Journal Articles

Integrated Genomics and Functional Validation Identifies a Global Kinase Gene Signature Impacting Genome Stability in Myeloma

Subodh Kumar, PhD, Leutz Buon, Srikanth Talluri, PhD, Chengcheng Liao, MD PhD, Jialan Shi, MD PhD, Mychell Neptune, Chandraditya Chakraborty, PhD, Mehmet Kemal Samur, PhD, Rao Prabhala, Masood A. Shammas, PhD, Nikhil C. Munshi, MD

Blood (2019) 134 (Supplement_1): 363.

https://doi.org/10.1182/blood-2019-129069

Published: 2019

Journal Articles

Preclinical Evaluation of ALLO-819, an Allogeneic CAR T Cell Therapy Targeting FLT3 for the Treatment of Acute Myeloid Leukemia

Cesar Sommer, PhD, Hsin-Yuan Cheng, PhD, Yik Andy Yeung, PhD, Duy Nguyen, Janette Sutton, Zea Melton, Julien Valton, PhD, Kris Poulsen, Ivana Djuretic, PhD, Thomas Van Blarcom, PhD, Javier Chaparro-Riggers, PhD, Barbra Sasu, PhD

Blood (2019) 134 (Supplement_1): 3921.

https://doi.org/10.1182/blood-2019-129025

Published: 2019

Journal Articles

Genome-Wide CRISPR Library Screening Identifies CDK6 As Genetic Vulnerability in Adult T-Cell Leukemia/Lymphoma

Takashi Ishio, Joji Shimono, Sarvesh Kumar, Yuquan Lin, Emmanuel Bachy, Michael N. Petrus, Yandan Yang, Michiyuki Maeda, Hideki Goto, Satoshi Hashino, Takanori Teshima, MD, Thomas A. Waldmann, MD, Louis Staudt, MD, Masao Nakagawa

Blood (2019) 134 (Supplement_1): 3781.

https://doi.org/10.1182/blood-2019-124670

Published: 2019

Journal Articles

Highly Efficient Multiplexed Base Editing with Minimized Off-Targets for the Development of Universal CAR-T Cells to Treat Pediatric T-ALL

Jason M Gehrke, PhD, Aaron Edwards, MS, Ryan C. Murray, MS, Amy Shaw, MS, Yeh-Chuin Poh, PhD, Sarah Smith, Yi Yu, PhD, Nicole M. Gaudelli, PhD, Waseem Qasim, MBBS, PhD, Giuseppe Ciaramella, PhD, B.Sc.

Blood (2019) 134 (Supplement_1): 5127.

https://doi.org/10.1182/blood-2019-130562

Published: 2019

Journal Articles

A Functional Analysis of SNP on Anthracycline-Induced Cardiotoxicity in a Uniform Genetic Background Using CRISPR/Cas9 Mediated Single-Base-Edited iPSCs

Takayuki Nakamura, MD, Shinichi Mizuno, MD PhD, Hidetoshi Ozawa, Yoshitaka Yamasaki, MD, Shuki Oya, MD, Satoshi Morishige, MD, Kazutoshi Aoyama, MD PhD, Maki Ymaguchi, MD PhD, Ritsuko Seki, PhD, Fumihiko Mouri, MD PhD, Koichi Osaki, MD, Koji Nagafuji, MD PhD

Blood (2019) 134 (Supplement_1): 5748.

https://doi.org/10.1182/blood-2019-123932

Published: 2019

Journal Articles

Universal' Fratricide-Resistant CAR T Cells Against T Cell Leukemia Generated By Coupled & Uncoupled Deamination Mediated Base Editing

Christos Georgiadis, PhD, Jane Rasaiyaah, PhD, Soragia Athina Gkazi, PhD, Roland Preece, Annie Etuk, PhD, Abraham Christi, Waseem Qasim, MBBS, PhD

Blood (2019) 134 (Supplement_1): 3219.

https://doi.org/10.1182/blood-2019-130057

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.

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

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

Journal Articles

Antibody Stabilization of Neutrophil Extracellular Trap-Platelet Factor 4 Complexes Is Therapeutic in a Murine Model of Endotoxemia

Amrita Sarkar, PhD, Gowthami Arepally, MD, M. Anna Kowalska, PhD, Douglas B. Cines, MD, Lubica Rauova, MD PhD, Mortimer Poncz, MD, Kandace Gollomp, MD

Blood (2018) 132 (Supplement 1): 271.

https://doi.org/10.1182/blood-2018-99-111381

Published: 2018

Journal Articles

Knockout of CBLB Greatly Enhances Anti-Tumor Activity of CAR T Cells

Kathryn Hooper, PhD, Kyle Havens, PhD, Anne-Rachel Krostag, BS, Michael S Magee, PhD, Unja Martin, Ankit Gupta, PhD, Yegor Smurnyy, PhD, Lisa Pechilis, Amanda Rode, Andrew Chavkin, MS, Shannon Grande, PhD, Richard A. Morgan, PhD, Jordan Jarjour, PhD, Alexander Astrakhan, PhD

Blood (2018) 132 (Supplement 1): 338.

https://doi.org/10.1182/blood-2018-99-119247

Published: 2018

NARROW

ILF2 Antisense Oligonucleotide Therapy and a CRISPR/Cas9-Based Screening for DNA Repair Effectors Identify Synthetic Lethal Approaches Enhancing Myeloma Cells Sensitivity to DNA Damage

Loss of METTL3 Mediated m6A RNA Modification Results in Double-Stranded RNA Induced Innate Immune Response and Hematopoietic Failure

Therapeutic Base Editing of Human Hematopoietic Stem Cells

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

Molecular Characterization and Novel Therapeutic Strategy for X-Linked Sideroblastic Anemia Associated with ALAS2 Missense Variants

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

Disease Modeling and Phenotype Rescue Using Inducible Pluripotent Stem Cells from Patients with Diamond-Blackfan Anemia

CRISPR/Cas9-Targeted De Novo DNA Methylation Is Maintained and Impacts the Colony Forming Potential of Human Hematopoietic CD34 + Cells

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

Polymorphisms in Inflammatory Genes Modulate the Occurrence of Clinical Complications in Patients with Sickle Cell Disease

Integrated Genomics and Functional Validation Identifies a Global Kinase Gene Signature Impacting Genome Stability in Myeloma

Preclinical Evaluation of ALLO-819, an Allogeneic CAR T Cell Therapy Targeting FLT3 for the Treatment of Acute Myeloid Leukemia

Genome-Wide CRISPR Library Screening Identifies CDK6 As Genetic Vulnerability in Adult T-Cell Leukemia/Lymphoma

Highly Efficient Multiplexed Base Editing with Minimized Off-Targets for the Development of Universal CAR-T Cells to Treat Pediatric T-ALL

A Functional Analysis of SNP on Anthracycline-Induced Cardiotoxicity in a Uniform Genetic Background Using CRISPR/Cas9 Mediated Single-Base-Edited iPSCs

Universal' Fratricide-Resistant CAR T Cells Against T Cell Leukemia Generated By Coupled & Uncoupled Deamination Mediated Base Editing

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

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

Antibody Stabilization of Neutrophil Extracellular Trap-Platelet Factor 4 Complexes Is Therapeutic in a Murine Model of Endotoxemia

Knockout of CBLB Greatly Enhances Anti-Tumor Activity of CAR T Cells

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American Society of Hematology

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American Society of Hematology

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