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Journal Articles

Hemoglobin S Polymerization Can be Inhibited By Alterations in Erythrocyte Glycolysis

William R. Simmons, Quan Li, PhD, John A. Hausman, Suilan Zheng, PhD, William Eaton, MD, Philip S. Low, PhD, David M. Bodine, PhD

Blood (2019) 134 (Supplement_1): 977.

https://doi.org/10.1182/blood-2019-121709

Published: 2019

Journal Articles

Concomitant Hydroxyurea and Voxelotor: Results from the HOPE Study

Russell E. Ware, MD PhD, Clark Brown, MD PhD, Mariane de Montalembert, MD PhD, Margaret Tonda, PharmD, Barbara Tong, PhD, Carolyn Hoppe, MD, Joshua Lehrer-Graiwer, MD MPhil, FACC, Miguel R. Abboud, MD

Blood (2019) 134 (Supplement_1): 1003.

https://doi.org/10.1182/blood-2019-130767

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.

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

Published: 2019

Journal Articles

Generation and Characterisation of KY1066, a Fully Human Antibody Targeting the Enzymatic Activity of Matriptase-2 for the Treatment of Iron Overload in Beta Thalassemia

Matthew Wake, PhD, Jonathan Papworth, BSc, Luke Bayliss, BSc, Benjamin Grimshaw, PhD, Natalie Rynkiewicz, PhD, Jemima Paterson, BSc, Alicia Poindron, MSc, Erin Spearing, Emily Carter, Robyne Hudson, BSc, Verena Petzer, MD, Amelie Largounez, BSc, Audrey Belot, BSc, Anais Palin, BSc, Igor Theurl, MD PhD, Volker Germaschewski, Delphine Meynard, PhD

Blood (2019) 134 (Supplement_1): 3532.

https://doi.org/10.1182/blood-2019-124075

Published: 2019

Journal Articles

Regulation of Iron Homeostasis By PTG-300 Improves Disease Parameters in Mouse Models for Beta-Thalassemia and Hereditary Hemochromatosis

Roopa Taranath, PhD, Gregory Bourne, PhD, Li Zhao, MD PhD, Brian Frederick, MS, Chelsea King, BA, David Liu, PhD

Blood (2019) 134 (Supplement_1): 3540.

https://doi.org/10.1182/blood-2019-129309

Published: 2019

Journal Articles

Development of a Personalized 3D Carotid Model for Cerebral Vasculopathy Monitoring in Sickle Cell Disease

Nguyen-Peyre Kim-Anh, PhD, Laurène Lenoir, Valentin Amar, Frédéric Segonds, Nuriye Akçay, Seguin Marion, Philippe Connes, PhD, France Pirenne, MD PhD, Jean-Frédéric Gerbeau, Irène Vignon-Clémentel, Suzanne Verlhac, MD, Pablo Bartolucci, MD PhD

Blood (2019) 134 (Supplement_1): 3570.

https://doi.org/10.1182/blood-2019-124327

Published: 2019

Journal Articles

Long Range Sequencing Shows Improved Resolution in the Detection of Beta Globin Cluster Variants

Aruna Rangan, MD, Molly S. Hein, Tejaswi Koganti, William G. Jenkinson, Christopher A. Hilker, Joseph H. Blommel, Tavanna R. Porter, MS, LCGC, Jin Jen, Nicole Hoppman, Rong He, MD, David S. Viswanatha, Ross Aleff, James D. Hoyer, MD, Eric Wieben, Eric W. Klee, PhD, Benjamin R. Kipp, Jennifer L. Oliveira, MD

Blood (2019) 134 (Supplement_1): 3548.

https://doi.org/10.1182/blood-2019-130652

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

Ex Vivo Expansion with E478 Increases the Rate of CRISPR-Mediated Homology Directed Repair of the Beta-Globin Gene in Human Hematopoietic Stem Cells By >10-Fold and Improves In Vivo Engraftment By >120-Fold

Kevin A. Goncalves, PhD, Megan D. Hoban, PhD, Sharon L. Hyzy, MS, Katia S. George, BA, Anthony E. Boitano, PhD, Michael P. Cooke, PhD

Blood (2019) 134 (Supplement_1): 4634.

https://doi.org/10.1182/blood-2019-131157

Published: 2019

Journal Articles

Increased Release of Soluble MD-2 in Sickle Cell Disease and Its Role in Pro-Inflammatory Signaling in Endothelial Cells

Ping Zhang, PhD, John D Belcher, PhD, Julia Nguyen, Fuad Abdulla, Gregory M Vercellotti, MD

Blood (2019) 134 (Supplement_1): 208.

https://doi.org/10.1182/blood-2019-122912

Published: 2019

Journal Articles

R-m-NHL-BFM-90/R-EPOCH Protocol in the Treatment of Patients with Primary Mediastinal Lymphoma: First Results

Nelly G Gabeeva, PhD, Darya A Koroleva, Svetlana A Tatarnikova, Anna K Smolyaninova, Svetlana Smirnova, MD PhD, Elena E Nikulina, Alla M. Kovrigina, MD PhD, Andrey B Sudarikov, PhD DSc, Eugene E. Zvonkov, MD PhD, Valery G. Savchenko, MD PhD academician

Blood (2019) 134 (Supplement_1): 5334.

https://doi.org/10.1182/blood-2019-127944

Published: 2019

Journal Articles

Genetic Engineering in Hematopoietic Stem Cells for Gene Therapy of Hemoglobinopathies

Giuliana Ferrari, PhD

Blood (2019) 134 (Supplement_1): SCI-23.

https://doi.org/10.1182/blood-2019-121119

Published: 2019

Journal Articles

Effects of Voxelotor (GBT440) Therapy on Laboratory Testing of Sickle Cell Patients

Michelle E. Savedra, MT(ASCP), Jessica L. Szuberski, MLS ASCP, Tavanna R. Porter, MS, LCGC, Molly S. Hein, Phuong L. Nguyen, MD, Min Shi, MD PhD, Aruna Rangan, MD, James D. Hoyer, MD, Jennifer L. Oliveira, MD

Blood (2019) 134 (Supplement_1): 1011.

https://doi.org/10.1182/blood-2019-131125

Published: 2019

Journal Articles

Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies

Laura Breda, PhD, Valentina Ghiaccio, PhD, Silvia Pires Lourenco, MSc, Danuta Jadwiga Jarocha, PhD, Yasuhiro Ikawa, MD PhD, Naoto Tanaka, PhD, Virginia Guzikowski, Carla Casu, PhD, Kim Smith-Whitley, MD, Janet L. Kwiatkowski, MD MSCE, Stefano Rivella, PhD

Blood (2019) 134 (Supplement_1): 2242.

https://doi.org/10.1182/blood-2019-124043

Published: 2019

Journal Articles

Combining HPFH Mutations in Human Adult HSCs to Enhance Reactivation of Fetal Hemoglobin

Nikoletta Psatha, Afrodite Georgakopoulou, PhDstudent, Chang Li, PhD, Pavel Sova, Hongjie Wang, PhD, Andre Lieber, MD PhD, Thalia Papayannopoulou, MD DrSci

Blood (2019) 134 (Supplement_1): 2246.

https://doi.org/10.1182/blood-2019-131487

Published: 2019

Journal Articles

Establishing a Link between Zinc Homeostasis and Globin Gene Expression

Oluwaseun Olaiya, DO, Halyna Fedosyuk, MS, Lesya V Novikova, PhD, Matthew P Parker, BS, Kenneth R Peterson, PhD

Blood (2019) 134 (Supplement_1): 3578.

https://doi.org/10.1182/blood-2019-130758

Published: 2019

Journal Articles

High Hemoglobin F in Sickle Cell Disease: Waning Protection with Age

Seda S. Tolu, MD, Morayma Reyes Gil, MD PhD, Ugochi Olivia Ogu, MD, Jui Choudhuri, MD, Roger Fecher, MD, Gracy Sebastian, NP, Caterina P. Minniti, MD

Blood (2019) 134 (Supplement_1): 3576.

https://doi.org/10.1182/blood-2019-122387

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

Chromatin Accessibility Mapping of Primary Erythroid Cell Populations Leads to Identification and Validation of Nuclear Factor I X (NFIX) As a Novel Fetal Hemoglobin (HbF) Repressor

Mudit Chaand, PhD, Chris Fiore, PhD, Brian T Johnston, MS, Diane H Moon, PhD, John P Carulli, PhD, Jeffrey R Shearstone, PhD

Blood (2019) 134 (Supplement_1): 812.

https://doi.org/10.1182/blood-2019-124337

Published: 2019

Journal Articles

Complementary Base Editing Approaches for the Treatment of Sickle Cell Disease and Beta Thalassemia

Ling Lin, PhD, Adrian P. Rybak, PhD, Conrad Rinaldi, M.S., Jonathan Yen, PhD, Yanfang Fu, PhD, Elsie Akrawi, Sarah Smith, Scott J. Haskett, M.S., Minerva E. Sanchez, Yeh-Chuin Poh, PhD, Michael S. Packer, PhD, Nicole M. Gaudelli, PhD, Manmohan Singh, PhD, Giuseppe Ciaramella, PhD, B.Sc., Dana N. Levasseur, PhD

Blood (2019) 134 (Supplement_1): 3352.

https://doi.org/10.1182/blood-2019-126710

Published: 2019

NARROW

Hemoglobin S Polymerization Can be Inhibited By Alterations in Erythrocyte Glycolysis

Concomitant Hydroxyurea and Voxelotor: Results from the HOPE Study

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

Generation and Characterisation of KY1066, a Fully Human Antibody Targeting the Enzymatic Activity of Matriptase-2 for the Treatment of Iron Overload in Beta Thalassemia

Regulation of Iron Homeostasis By PTG-300 Improves Disease Parameters in Mouse Models for Beta-Thalassemia and Hereditary Hemochromatosis

Development of a Personalized 3D Carotid Model for Cerebral Vasculopathy Monitoring in Sickle Cell Disease

Long Range Sequencing Shows Improved Resolution in the Detection of Beta Globin Cluster 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

Ex Vivo Expansion with E478 Increases the Rate of CRISPR-Mediated Homology Directed Repair of the Beta-Globin Gene in Human Hematopoietic Stem Cells By >10-Fold and Improves In Vivo Engraftment By >120-Fold

Increased Release of Soluble MD-2 in Sickle Cell Disease and Its Role in Pro-Inflammatory Signaling in Endothelial Cells

R-m-NHL-BFM-90/R-EPOCH Protocol in the Treatment of Patients with Primary Mediastinal Lymphoma: First Results

Genetic Engineering in Hematopoietic Stem Cells for Gene Therapy of Hemoglobinopathies

Effects of Voxelotor (GBT440) Therapy on Laboratory Testing of Sickle Cell Patients

Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies

Combining HPFH Mutations in Human Adult HSCs to Enhance Reactivation of Fetal Hemoglobin

Establishing a Link between Zinc Homeostasis and Globin Gene Expression

High Hemoglobin F in Sickle Cell Disease: Waning Protection with Age

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

Chromatin Accessibility Mapping of Primary Erythroid Cell Populations Leads to Identification and Validation of Nuclear Factor I X (NFIX) As a Novel Fetal Hemoglobin (HbF) Repressor

Complementary Base Editing Approaches for the Treatment of Sickle Cell Disease and Beta Thalassemia

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American Society of Hematology

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ASH Publications

American Society of Hematology

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