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Journal Articles

β-Hemoglobinopathies lead the way

Yogen Saunthararajah

Blood (2021) 137 (12): 1567–1569.

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

Published: 2021

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.

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

Published: 2021

Includes: Supplemental data

Journal Articles

Targeting the bone marrow niche in hemoglobinopathies

Nadia Carlesso

Blood (2020) 136 (5): 529–531.

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.

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.

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

Published: 2019

Journal Articles

Transcranial Doppler Screening in Children with Sickle Cell Anemia Is Feasible in Central India and Reveals High Risk of Stroke

Dipty Jain, MD, Kavitha Ganesan, MBBS, Sati Sahota, Deepika S. Darbari, MD, Lakshmanan Krishnamurti, MD, Fenella Jane Kirkham, MD

Blood (2019) 134 (Supplement_1): 2279.

https://doi.org/10.1182/blood-2019-131416

Published: 2019

Journal Articles

Development of a Severity Score System for Thalassemia Syndromes

Blood (2019) 134 (Supplement_1): 2225.

https://doi.org/10.1182/blood-2019-124675

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

Comparison of Clinical Evolution of Children with Sickle Cell Disease before and after Treatment with Hydroxyurea at Saint Damien Hospital, Tabarre-Haiti,2013-2018

Nora St Victor Dély, MD, Ofelia A. Alvarez, MD, Vanessa J Dor, MD, Emmeline Lerebours, MD

Blood (2019) 134 (Supplement_1): 2306.

https://doi.org/10.1182/blood-2019-124105

Published: 2019

Journal Articles

Phenotypic Corrections of Ex Vivo Erythrocytes Derived from Chronic Hemolytic Anemia Patients Due to Krϋppel-like Factor 1 ( KLF-1 ) Mutations By AG-348: A Novel Synthetic Pyruvate Kinase Activator

Thidarat Suksangpleng, PhD, Kalaya Tachavanich, M.Sc., Suchada Riolueang, MSc, Supachai Ekwattanakit, PhDMD, Vip Viprakasit, MD DPhil

Blood (2019) 134 (Supplement_1): 2220.

https://doi.org/10.1182/blood-2019-131720

Published: 2019

Journal Articles

Allo-Immunisation in Sickle Cell Patients in a Large British Specialist Haemoglobinopathy Centre

Sarrah Tayabali, MBBS, BSc, DipRCPath, Karen Madgwick, FIBMS, CSci, DMLM, PgDip, Arne de Kreuk, MD PhD

Blood (2019) 134 (Supplement_1): 2451.

https://doi.org/10.1182/blood-2019-130492

Published: 2019

Journal Articles

Proteomic Discovery: Elevated Neurogranin Levels in Children with Sickle Cell Disease

Eboni I. Lance, MD, Lisa Faulcon, MD, Zongming Fu, PhD, Jun Yang, PhD, Donna Whyte, MD, John J. Strouse, MD, PhD, Emily Barron-Casella, PhD, Kimberly Jones, Jennifer Van Eck, PhD, James F. Casella, MD, Allen Everett, MD

Blood (2019) 134 (Supplement_1): 2296.

https://doi.org/10.1182/blood-2019-122015

Published: 2019

Journal Articles

Prenatal Hemoglobinopathy Screening Practices: Areas for Improvement

Scott Moerdler, MD, Ellen Fraint, MD, Ellen Silver, PhD, Siobhan M Dolan, MD, M.P.H., Kafui A Demasio, MD, M.P.H., Michael E. Roth, MD, Deepa Manwani, MD, Kerry A Morrone, MD

Blood (2019) 134 (Supplement_1): 2298.

https://doi.org/10.1182/blood-2019-126084

Published: 2019

Journal Articles

Investigation of Cell-Contact Dependent Alterations in Immune Synapse Formation between T Cells and BCP-ALL Cells

Britta Wenske, PhD, Lothar Marischen, PhD, Julia Welzenbach, André Heimbach, PhD, Per Hoffmann, PhD, Selim Corbacioglu, MD, David A Williams, MD, Anja Troeger, MD

Blood (2019) 134 (Supplement_1): 2754.

https://doi.org/10.1182/blood-2019-128835

Published: 2019

Journal Articles

Untargeted Metabolomics on Dried Blood Spots for the Diagnosis and Clinical Follow up of Rare Hereditary Anemias

Birgit van Dooijeweert, MD, Melissa Broeks, Msc, Judith Jans, PhD, Eduard J. van Beers, MD PhD, Nanda Verhoeven, PhD, Richard van Wijk, PhD, Marije Bartels, MD PhD

Blood (2019) 134 (Supplement_1): 3376.

https://doi.org/10.1182/blood-2019-131693

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

Journal Articles

Fully Closed, Large-Scale, and Clinical Grade Cell Sorting of Hematopoietic Stem Cell (HSC)-Enriched CD90 + Cells for Transplantation and Gene Therapy

Stefan Radtke, PhD, Margaret Cui, Anai M Perez, Yan-Yi Chan, Stefanie Schmuck, BS, Andrew J Berger, PhD, Thomas Eunson, BSc, Hans-Peter Kiem, MD PhD

Blood (2019) 134 (Supplement_1): 3246.

https://doi.org/10.1182/blood-2019-125969

Published: 2019

Journal Articles

Results of a Multicentre, Randomized, Controlled Open-Label Study on the Use of Anti-T-Lymphocyte Globulin (ATLG) and Rituximab for Immunomodulation of Graft-Versus-Host Disease (GvHD) and Graft Failure (GF) in Patients with Non-Malignant Disorders

Mattia Algeri, MD, Stefania Galimberti, PhD, Maria Ester Bernardo, MD PhD, Attilio Rovelli, MD, Marco Zecca, MD, Giorgio La Nasa, MD, Sarah Marktel, MD, Pietro Merli, MD, Alice Bertaina, MD PhD, Daria Pagliara, MD PhD, Emilia Boccieri, MD, Francesca Del Bufalo, MD, Stefania Gaspari, MD, Giulia Capitoli, Maria Grazia Valsecchi, PhD, Franco Locatelli, MD PhD

Blood (2019) 134 (Supplement_1): 3280.

https://doi.org/10.1182/blood-2019-128925

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

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β-Hemoglobinopathies lead the way

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

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

Transcranial Doppler Screening in Children with Sickle Cell Anemia Is Feasible in Central India and Reveals High Risk of Stroke

Development of a Severity Score System for Thalassemia Syndromes

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

Comparison of Clinical Evolution of Children with Sickle Cell Disease before and after Treatment with Hydroxyurea at Saint Damien Hospital, Tabarre-Haiti,2013-2018

Phenotypic Corrections of Ex Vivo Erythrocytes Derived from Chronic Hemolytic Anemia Patients Due to Krϋppel-like Factor 1 ( KLF-1 ) Mutations By AG-348: A Novel Synthetic Pyruvate Kinase Activator

Allo-Immunisation in Sickle Cell Patients in a Large British Specialist Haemoglobinopathy Centre

Proteomic Discovery: Elevated Neurogranin Levels in Children with Sickle Cell Disease

Prenatal Hemoglobinopathy Screening Practices: Areas for Improvement

Investigation of Cell-Contact Dependent Alterations in Immune Synapse Formation between T Cells and BCP-ALL Cells

Untargeted Metabolomics on Dried Blood Spots for the Diagnosis and Clinical Follow up of Rare Hereditary Anemias

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

Fully Closed, Large-Scale, and Clinical Grade Cell Sorting of Hematopoietic Stem Cell (HSC)-Enriched CD90 + Cells for Transplantation and Gene Therapy

Results of a Multicentre, Randomized, Controlled Open-Label Study on the Use of Anti-T-Lymphocyte Globulin (ATLG) and Rituximab for Immunomodulation of Graft-Versus-Host Disease (GvHD) and Graft Failure (GF) in Patients with Non-Malignant Disorders

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

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American Society of Hematology

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