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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

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

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

Evaluating Luspatercept Responders in the Phase 3, Randomized, Double-Blind, Placebo-Controlled BELIEVE Trial of Luspatercept in Adult Beta-Thalassemia Patients (Pts) Who Require Regular Red Blood Cell (RBC) Transfusions

Blood (2019) 134 (Supplement_1): 3545.

https://doi.org/10.1182/blood-2019-122685

Published: 2019

Journal Articles

Luspatercept Significantly Reduces Red Blood Cell (RBC) Transfusion Burden, Regardless of Gene Mutation Frequency, Spectrum, and Prognostic Significance, Among Patients (Pts) with LR-MDS Enrolled in the MEDALIST Trial

Blood (2019) 134 (Supplement_1): 2999.

https://doi.org/10.1182/blood-2019-123655

Published: 2019

Journal Articles

A Phase 2 Study of Luspatercept in Patients with Myelofibrosis-Associated Anemia

Blood (2019) 134 (Supplement_1): 557.

https://doi.org/10.1182/blood-2019-122546

Published: 2019

Journal Articles

Sickle Cell Disease Burden in North Lebanon

Adlette Inati, MD, Chadi Al Alam, Cristel El Ojaimi, Taghrid Hamad, Hemanth Kanakamedala, Virginia Pilipovic, Ramzieh Sabah

Blood (2019) 134 (Supplement_1): 1022.

https://doi.org/10.1182/blood-2019-128311

Published: 2019

Journal Articles

Evaluation of Pulmonary Function in Patient with Transfusion Dependent Beta Thalassemia with Severe Iron Overload

Mohamed A Yassin, MBBS, MSc, Mushtaq Ahmad, MD, Shehab Mohamed, MD, Khadra S Yassin, BSC, Abdulqadir Jeprel Nashwan, BSN, MSN, Vincenzo Desanctis, MD PhD, Ashraf Tawfiq Soliman, MD PhD

Blood (2019) 134 (Supplement_1): 4818.

https://doi.org/10.1182/blood-2019-121828

Published: 2019

Journal Articles

Non Invasive Evaluation of Bone Marrow Activity in Patients with Sickle Cell Disease: Correlation with Disease Features, Genotype, Markers of Erythropoiesis, Iron Metabolism and Hydroxyurea Treatment

Ioannis Papassotiriou, PhD, Pagona Flevari, MD, Christos Poziopoulos, MD, Sofia Zaliou, PhD, Vasilis Tsaousis, PhD, Katerina Larissi, MD, Maria Dimopoulou, MD, Veroniki Komninaka, MD, Marianna Politou, MD, Evangelos Terpos, MD, Ersi Voskaridou

Blood (2019) 134 (Supplement_1): 4821.

https://doi.org/10.1182/blood-2019-121622

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

Assessment of Longer-Term Efficacy and Safety in the Phase 3, Randomized, Double-Blind, Placebo-Controlled MEDALIST Trial of Luspatercept to Treat Anemia in Patients (Pts) with Revised International Prognostic Scoring System (IPSS-R) Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS) with Ring Sideroblasts (RS) Who Require Red Blood Cell (RBC) Transfusions

Blood (2019) 134 (Supplement_1): 841.

https://doi.org/10.1182/blood-2019-123064

Published: 2019

Journal Articles

CRISPR/dCas9 (D10A) -Mediated Hb CS Gene Editing and Identification of Genetically Modified Fibroblasts

Lian Yu, Wei Hao Wu, WANG Shunqing, Ken H. Young, Funeng Jiang, PhD

Blood (2019) 134 (Supplement_1): 5754.

https://doi.org/10.1182/blood-2019-129931

Published: 2019

Journal Articles

Hematologic Improvement-Neutrophil and -Platelet in the MEDALIST Trial: Multilineage Data from a Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Luspatercept to Treat Anemia in Patients with Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS) with Ring Sideroblasts (RS) Who Require Red Blood Cell (RBC) Transfusions

Guillermo Garcia-Manero, MD, Ghulam J. Mufti, Pierre Fenaux, MD PhD, Rena Buckstein, MD FRCPC, Valeria Santini, MD, María Díez-Campelo, MD PhD, Carlo Finelli, Mario Cazzola, MD, Osman Ilhan, Mikkael A. Sekeres, MD MS, Alan F. List, MD, Abderrahmane Laadem, Rodrigo Ito, Jennie Zhang, Anita Rampersad, Daniel Sinsimer, Peter G. Linde, Uwe Platzbecker, MD, Rami Komrokji

Blood (2019) 134 (Supplement_1): 4243.

https://doi.org/10.1182/blood-2019-123048

Published: 2019

Journal Articles

Greater Red Blood Cell Transfusion Burden Is Associated with More Healthcare Resource Utilization in Patients with Beta-Thalassemia

Chao-Hsiun Tang, Wesley Furnback, Bruce C.M. Wang, Jackson Tang, Vicky Wei-Hsuen Huang, Derek Tang, PhD BSPharm, Meng-Yao Lu, Khaled M. Musallam

Blood (2019) 134 (Supplement_1): 5790.

https://doi.org/10.1182/blood-2019-122747

Published: 2019

Journal Articles

Ex vivo Engineering of Red Blood Cells

Jan Frayne, BsC, PhD, Deborah E Daniels, Jan Frayne, BsC PhD, Timothy J Satchwell, PhD, Joseph Hawksworth, BSc PhD, Belinda K Singleton, Tatyana N Andrienko, PhD, Marieangela C Wilson, Sabine Kupzig, Marjolein Meinders, Ryo Kurita, PhD, Yukio Nakamura, MD PhD, Ivan Ferrer Vicens, PhD MSc, BSc, Rebecca Griffiths, PhD, Nicola Cogan, Kongtana Trakarnsanga, MD PhD, David J. Anstee, PhD FRCPath, Ashley Mark Toye, PhD

Blood (2019) 134 (Supplement_1): SCI-5.

https://doi.org/10.1182/blood-2019-121117

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

Implementation of a Standard Order Set at a Sickle Cell Acute Care Observation Unit

Renee Cheng, MD, Rishi Patel, MD, Sandra Kang, MD, Frances Tian, MD, Xu Zhang, PhD, Jin Han, PharmD, PhD, Jayleen Patel, MD, Netanel Zilberstein, MD, Victor R Gordeuk, MD, Michel Gowhari, DO

Blood (2019) 134 (Supplement_1): 3396.

https://doi.org/10.1182/blood-2019-130877

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

Reduced Number and Function of Endothelial Progenitor Cells in Patients with Beta Thalassemia Major

Galia Spectre, Pinhas Stark, MD, Arza Steimatzky, MD, Pia Raanani, MD, Dorit Lev, PhD, Ran Kornowski, MD, Idit Pazgal, MD

Blood (2019) 134 (Supplement_1): 2247.

https://doi.org/10.1182/blood-2019-129851

Published: 2019

NARROW

Therapeutic Base Editing of Human Hematopoietic Stem Cells

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

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

Evaluating Luspatercept Responders in the Phase 3, Randomized, Double-Blind, Placebo-Controlled BELIEVE Trial of Luspatercept in Adult Beta-Thalassemia Patients (Pts) Who Require Regular Red Blood Cell (RBC) Transfusions

Luspatercept Significantly Reduces Red Blood Cell (RBC) Transfusion Burden, Regardless of Gene Mutation Frequency, Spectrum, and Prognostic Significance, Among Patients (Pts) with LR-MDS Enrolled in the MEDALIST Trial

A Phase 2 Study of Luspatercept in Patients with Myelofibrosis-Associated Anemia

Sickle Cell Disease Burden in North Lebanon

Evaluation of Pulmonary Function in Patient with Transfusion Dependent Beta Thalassemia with Severe Iron Overload

Non Invasive Evaluation of Bone Marrow Activity in Patients with Sickle Cell Disease: Correlation with Disease Features, Genotype, Markers of Erythropoiesis, Iron Metabolism and Hydroxyurea Treatment

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

CRISPR/dCas9 (D10A) -Mediated Hb CS Gene Editing and Identification of Genetically Modified Fibroblasts

Greater Red Blood Cell Transfusion Burden Is Associated with More Healthcare Resource Utilization in Patients with Beta-Thalassemia

Ex vivo Engineering of Red Blood Cells

Genetic Engineering in Hematopoietic Stem Cells for Gene Therapy of Hemoglobinopathies

Implementation of a Standard Order Set at a Sickle Cell Acute Care Observation Unit

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

Reduced Number and Function of Endothelial Progenitor Cells in Patients with Beta Thalassemia Major

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American Society of Hematology

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ASH Publications

American Society of Hematology

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