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Journal Articles

A natural DNMT1 mutation elevates the fetal hemoglobin level via epigenetic derepression of the γ-globin gene in β-thalassemia

Yi Gong, Xinhua Zhang, Qianqian Zhang, Yanxia Zhang, Yuhua Ye, Wenxia Yu, Congwen Shao, Tizhen Yan, Jin Huang, Jianmei Zhong, Li Wang, Yaoyun Li, Liren Wang, Xiangmin Xu

Blood (2021) 137 (12): 1652–1657.

DOI: https://doi.org/10.1182/blood.2020006425

Published: 2021

Includes: Supplemental data

Journal Articles

β-Hemoglobinopathies lead the way

Yogen Saunthararajah

Blood (2021) 137 (12): 1567–1569.

DOI: https://doi.org/10.1182/blood.2020009961

Published: 2021

Journal Articles

Targeting the bone marrow niche in hemoglobinopathies

Nadia Carlesso

Blood (2020) 136 (5): 529–531.

DOI: https://doi.org/10.1182/blood.2020006305

Published: 2020

Journal Articles

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

Erica B. Esrick, MD, Maureen Achebe, MD MPH, Myriam Armant, PhD, Pablo Bartolucci, MD PhD, Marioara Felicia Ciuculescu, MD, Heather Daley, Colleen Dansereau, MSN, RN, CPN, Giuseppe Di Caprio, PhD, Bronner Goncalves, MD PhD, Nicolas Hebert, Matthew M Heeney, MD, John M. Higgins, MD PhD, Leslie E. Lehmann, MD, John P Manis, MD, Olivier Negre, PhD, Sarah Nikiforow, MD PhD, Ethan Schonbrun, PhD, David K. Wood, PhD, David A. Williams, MD

Blood (2019) 134 (Supplement_2): LBA-5.

DOI: https://doi.org/10.1182/blood-2019-132745

Published: 2019

Journal Articles

Heme-regulated eIF2α kinase in erythropoiesis and hemoglobinopathies

Jane-Jane Chen, Shuping Zhang

Blood (2019) 134 (20): 1697–1707.

DOI: https://doi.org/10.1182/blood.2019001915

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.

DOI: 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.

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

Published: 2019

Journal Articles

Validation of Hemolytic Anemia Codes in the French Hospital Database

Julien Maquet, Hélène Derumeaux, MD PhD, Maryse Lapeyre-Mestre, MD PhD, Laurent Sailler, MD PhD, Guillaume Moulis, MD PhD

Blood (2019) 134 (Supplement_1): 3460.

DOI: https://doi.org/10.1182/blood-2019-124697

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.

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

Published: 2019

Journal Articles

Potential Impact of Sickle Hemoglobin Concentration on Survival Among Patients with Renal Medullary Carcinoma and Sickle Cell Trait

Marcus A. Carden, MD, Jonathan Metts, MD, John M. McCarty, MD, Sarah Mitchell, MD, Bradley Carthon, MD PhD, David A. Siegel, MD, Louis Rapkin, MD, Morgan L. McLemore, MD

Blood (2019) 134 (Supplement_1): 4843.

DOI: https://doi.org/10.1182/blood-2019-125088

Published: 2019

Journal Articles

Downregulation of Transcription Factor LRF/ZBTB7A Increases Fetal Hemoglobin Expression in β-Thalassemia/Hemoglobin E Erythroid Cells

Sukanya Chumchuen, MSc, BSc, Tanapat Pornsukjantra, B.Sc., Pinyaphat Khamphikham, PhD, Usanarat Anurathapan, MD, Orapan Sripichai, PhD, Duantida Songdej, Suradej Hongeng, MD

Blood (2019) 134 (Supplement_1): 3549.

DOI: https://doi.org/10.1182/blood-2019-126664

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.

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

Published: 2019

Journal Articles

Evaluation of Newborns Screening Laboratory Tests for Sickle Cell Disease and Other Haemoglobinopathies in Tanzania

Heavenlight Hebron Christopher, BSc, Adam Burns, BSc, PhD, Emmanuel Josephat, BSc, Sephord Saul, BSc, Josephine Mgaya, BSc, Julie Makani, MD PhD FRCP, Siana Watoky Nkya, PhD MSc, BSc, Anna Schuh, PhD

Blood (2019) 134 (Supplement_1): 4817.

DOI: https://doi.org/10.1182/blood-2019-129900

Published: 2019

Journal Articles

Ethical Issues and Barriers for Multi-National Paediatric Clinical Trials: The Challenging Experience of the DEEP Project

Viviana Giannuzzi, PhD, Mariagrazia Felisi, PhD, Hugo Devlieger, Aurelio Maggio, MD, George Papanikolaou, MD PhD, Giorgio Reggiardo, Bianca Tempesta, PhD, Fernando Tricta, MD, Donato Bonifazi

Blood (2019) 134 (Supplement_1): 4820.

DOI: https://doi.org/10.1182/blood-2019-124689

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.

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

Published: 2019

Journal Articles

Validity of HbA1c in Diagnosing Diabetes Among People with Sickle Cell Trait in Tanzania

Belinda Kweka, MD, Eric Lyimo, BSc, Jeremiah Kidola, PhD, Suzanne Filteau, PhD, Henrik Friis, PhD, Alphaxard Manjurano, Daniel Faurholt Jepsen, PhD, George PrayGod, MD, Heimburger Douglas C, MD

Blood (2019) 134 (Supplement_1): 4852.

DOI: https://doi.org/10.1182/blood-2019-125326

Published: 2019

Journal Articles

Greatalys: Generating Real World Evidence across Italy in Sickle Cell Disease

Chiara Castiglioni, Claudia Condorelli, Paola Amore, Valentina Perrone, Davide Alessandrini, Chiara Veronesi, Luca Degli Esposti, Eleonora Premoli, Carina Fiocchi

Blood (2019) 134 (Supplement_1): 4707.

DOI: https://doi.org/10.1182/blood-2019-125612

Published: 2019

Journal Articles

Impact of Human Leukocyte Antigen on the Risk of Stroke in Patients with Sickle Cell Disease

Huda Al-Harrasi, MD, Murtadha Al-Khabori, MD, Salam Al-Kindi, MD, Yasser Ahmed Mohamed Soliman Wali, MD, Mohamed Al Huneini, MD, Hamad Al-Riyami, PhD

Blood (2019) 134 (Supplement_1): 4831.

DOI: https://doi.org/10.1182/blood-2019-125038

Published: 2019

Journal Articles

Neurocognitive Testing in Pediatric Sickle Cell Disease: Full Scale IQ Assessment May Not be the Full Story

Shelby Gruntorad, MSc, Jasmin Searcy, PhD, Radhika Peddinti, MD

Blood (2019) 134 (Supplement_1): 4833.

DOI: https://doi.org/10.1182/blood-2019-129178

Published: 2019

Journal Articles

Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Blood (2019) 134 (Supplement_1): 4629.

DOI: https://doi.org/10.1182/blood-2019-126161

Published: 2019

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NARROW

A natural DNMT1 mutation elevates the fetal hemoglobin level via epigenetic derepression of the γ-globin gene in β-thalassemia

β-Hemoglobinopathies lead the way

Targeting the bone marrow niche in hemoglobinopathies

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

Heme-regulated eIF2α kinase in erythropoiesis and hemoglobinopathies

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

Validation of Hemolytic Anemia Codes in the French Hospital Database

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

Potential Impact of Sickle Hemoglobin Concentration on Survival Among Patients with Renal Medullary Carcinoma and Sickle Cell Trait

Downregulation of Transcription Factor LRF/ZBTB7A Increases Fetal Hemoglobin Expression in β-Thalassemia/Hemoglobin E Erythroid Cells

Establishing a Link between Zinc Homeostasis and Globin Gene Expression

Evaluation of Newborns Screening Laboratory Tests for Sickle Cell Disease and Other Haemoglobinopathies in Tanzania

Ethical Issues and Barriers for Multi-National Paediatric Clinical Trials: The Challenging Experience of the DEEP Project

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

Validity of HbA1c in Diagnosing Diabetes Among People with Sickle Cell Trait in Tanzania

Greatalys: Generating Real World Evidence across Italy in Sickle Cell Disease

Impact of Human Leukocyte Antigen on the Risk of Stroke in Patients with Sickle Cell Disease

Neurocognitive Testing in Pediatric Sickle Cell Disease: Full Scale IQ Assessment May Not be the Full Story

Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

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