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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

Brief Report
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
Journal: Blood
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
Journal: Blood
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
Journal: Blood
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
Journal: Blood
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
Journal: Blood
Blood (2019) 134 (20): 1697–1707.
DOI: https://doi.org/10.1182/blood.2019001915
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
Journal: Blood
Blood (2019) 134 (Supplement_1): 2451.
DOI: https://doi.org/10.1182/blood-2019-130492
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
Journal: Blood
Blood (2019) 134 (Supplement_1): 2242.
DOI: 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
Journal: Blood
Blood (2019) 134 (Supplement_1): 2246.
DOI: 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
Journal: Blood
Blood (2019) 134 (Supplement_1): 2306.
DOI: 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
Journal: Blood
Blood (2019) 134 (Supplement_1): 2220.
DOI: https://doi.org/10.1182/blood-2019-131720
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
Journal: Blood
Blood (2019) 134 (Supplement_1): 3352.
DOI: https://doi.org/10.1182/blood-2019-126710
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
Journal: Blood
Blood (2019) 134 (Supplement_1): 2754.
DOI: https://doi.org/10.1182/blood-2019-128835
Published: 2019
Journal Articles

Durable and Robust Fetal Globin Induction without Anemia in Rhesus Monkeys Following Autologous Hematopoietic Stem Cell Transplant with BCL11A Erythroid Enhancer Editing

Selami Demirci, PhD, Jing Zeng, MD, Yuxuan Wu, PhD, Naoya Uchida, MD, Jackson Gamer, Morgan Yapundich, Claire Drysdale, Aylin C. Bonifacino, Allen E. Krouse, Nathaniel S. Linde, Robert E. Donahue, VMD, Anne H. Shen, Juan J. Haro-Mora, PhD, Alexis Leonard, MD, Tina Nassehi, Kevin Luk, Scot A. Wolfe, Cicera R. Lazzarotto, Shengdar Q. Tsai, PhD, Mitchell J. Weiss, MD, PhD, Daniel E. Bauer, MD PhD, John F. Tisdale, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4632.
DOI: https://doi.org/10.1182/blood-2019-129394
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
Journal: Blood
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

Vip Viprakasit, Ali Taher, Olivier Hermine, John Porter, Antonio Piga, Kevin H. M. Kuo, MDMSc,FRCPC, Thomas Coates, MD, Ersi Voskaridou, Hong Keng Liew, Silverio Perrotta, Abderrahim Khelif, Antonis Kattamis, Abderrahmane Laadem, Jeevan K. Shetty, Wen-Ling Kuo, Dimana Miteva, Tatiana Zinger, Daniel Sinsimer, Peter G. Linde, Maria Domenica Cappellini, MD
Journal: Blood
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
Journal: Blood
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
Journal: Blood
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
Journal: Blood
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
Journal: Blood
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
Journal: Blood
Blood (2019) 134 (Supplement_1): 3578.
DOI: https://doi.org/10.1182/blood-2019-130758
Published: 2019