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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.

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

Published: 2021

Includes: Supplemental data

Journal Articles

Less (Fe) is more (Hb) in SCA

Carlo Brugnara

Blood (2021) 137 (11): 1446–1447.

https://doi.org/10.1182/blood.2020010131

Published: 2021

Journal Articles

Hematopoietic stem cell function in β-thalassemia is impaired and is rescued by targeting the bone marrow niche

Annamaria Aprile, Alessandro Gulino, Mariangela Storto, Isabella Villa, Stefano Beretta, Ivan Merelli, Alessandro Rubinacci, Maurilio Ponzoni, Sarah Marktel, Claudio Tripodo, Maria Rosa Lidonnici, Giuliana Ferrari

Blood (2020) 136 (5): 610–622.

https://doi.org/10.1182/blood.2019002721

Published: 2020

Includes: Supplemental data

Journal Articles

Antibodies against the erythroferrone N-terminal domain prevent hepcidin suppression and ameliorate murine thalassemia

João Arezes, Niall Foy, Kirsty McHugh, Doris Quinkert, Susan Benard, Anagha Sawant, Joe N. Frost, Andrew E. Armitage, Sant-Rayn Pasricha, Pei Jin Lim, May S. Tam, Edward Lavallie, Debra D. Pittman, Orla Cunningham, Matthew Lambert, John E. Murphy, Simon J. Draper, Reema Jasuja, Hal Drakesmith

Blood (2020) 135 (8): 547–557.

https://doi.org/10.1182/blood.2019003140

Published: 2020

Includes: Supplemental data

Journal Articles

Single-cell analysis of bone marrow–derived CD34 + cells from children with sickle cell disease and thalassemia

Peng Hua, Noemi Roy, Josu de la Fuente, Guanlin Wang, Supat Thongjuea, Kevin Clark, Anindita Roy, Bethan Psaila, Neil Ashley, Yvonne Harrington, Claus Nerlov, Suzanne M. Watt, Irene Roberts, James O. J. Davies

Blood (2019) 134 (23): 2111–2115.

https://doi.org/10.1182/blood.2019002301

Published: 2019

Includes: Supplemental data

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

Correlation between Changes in Cardiac Iron and Hepatic Iron in Pediatric Patients with Thalassemia Major

Alessia Pepe, MD, Antonella Meloni, PhD, Aldo Filosa, Laura Pistoia, Tommaso Casini, Roberto Lisi, Maria Caterina Putti, MD, Aurelio Maggio, MD, Calogera Gerardi, MD, Priscilla Fina, Vincenzo Positano, Maddalena Casale, MD

Blood (2019) 134 (Supplement_1): 2241.

https://doi.org/10.1182/blood-2019-121730

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

Cerebral Oxygen Delivery and Metabolic Rate in Chronically Anemic Subjects

Chau Vu, BS, Adam Bush, PhD, Thomas Coates, MD, John C Wood, MD PhD

Blood (2019) 134 (Supplement_1): 2273.

https://doi.org/10.1182/blood-2019-125897

Published: 2019

Journal Articles

2'-O-Methoxyethyl Splice-Switching Oligos to Reverse Splicing from IVS2-745 β-Thalassemia Patient Cells: A Foundation for Potential Therapies

Blood (2019) 134 (Supplement_1): 2244.

https://doi.org/10.1182/blood-2019-127017

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

Journal Articles

The Oxygenscan Provides Clinically Relevant Biomarkers for Treatment Efficacy That Are Associated with Frequency of Vaso-Occlusive Crisis in Sickle Cell Disease

Minke A.E. Rab, MD, Richard van Wijk, PhD, Celeste K. Kanne, BA, Brigitte A. van Oirschot, Jennifer Bos, Camille Boisson, Maite E. Houwing, MD, Jorn J. Gerritsma, MD, Karin Fijnvandraat, MD PhD, Celine Renoux, PhD, Marije Bartels, MD PhD, Erfan Nur, MD PhD, Marjon H. Cnossen, MD PhD, Philippe Joly, PhD, Romain Fort, MD, Philippe Connes, PhD, Eduard J. van Beers, MD PhD, Vivien A. Sheehan, MD PhD

Blood (2019) 134 (Supplement_1): 2275.

https://doi.org/10.1182/blood-2019-128870

Published: 2019

Journal Articles

Opioid Analgesics Are Associated with Albuminuria in Adult Patients with Sickle Cell Anemia

Ashley Thrower, BS, Zhou Laura, Vimal K. Derebail, MD, David Wichlan, Ashley Smith, RN, Jianwen Cai, PhD, Kenneth I. Ataga, MD

Blood (2019) 134 (Supplement_1): 2308.

https://doi.org/10.1182/blood-2019-131368

Published: 2019

Journal Articles

A Longitudinal Study in Elderly Patients with Non-Transfusion Dependent Thalassemia (NTDT) Emphasizes the Size of Splenomegaly Predicts Transfusion Requirement Later in Life

Supachai Ekwattanakit, PhDMD, Vip Viprakasit, MD DPhil

Blood (2019) 134 (Supplement_1): 2250.

https://doi.org/10.1182/blood-2019-122296

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

Factor-Associated Risk Factors of Mild Cognitive Impairment in Thalassemia Patients : Probable Role of FGF21

Wasan Theerajangkhaphichai, MD, Adisak Tantiworawit, MD, Jirapas Sripetchwandee, PhD, Nattayaporn Apaijai, PhD, Sirawit Sriwichaiin, MD, Napatsorn Saiyasit, Kewarin Jinawong, Busarin Arunsak, Pokpong Piriyakhuntorn, MD, Thanawat Rattanathammethee, MD, Sasinee Hantrakool, MD, Chatree Chai-Adisaksopha, MD PhD, Ekarat Rattarittamrong, MD, Lalita Norasetthada, MD, Pimlak Charoenkwan, MD, Nipon Chattipakorn, MD PhD, Siriporn Chattipakorn, DDS, PhD

Blood (2019) 134 (Supplement_1): 2251.

https://doi.org/10.1182/blood-2019-125182

Published: 2019

Journal Articles

Effects of Luspatercept on Iron Overload and Impact on Responders to Luspatercept: Results from the BELIEVE Trial

John Porter, Maria Domenica Cappellini, MD, Thomas Coates, MD, Olivier Hermine, Vip Viprakasit, MD DPhil, Ersi Voskaridou, Hong Keng Liew, Silverio Perrotta, Abderrahim Khelif, Antonis Kattamis, Abderrahmane Laadem, Jeevan K. Shetty, Wen-Ling Kuo, Dimana Miteva, Tatiana Zinger, Peter G. Linde, Daniel Sinsimer, Ali Taher

Blood (2019) 134 (Supplement_1): 2245.

https://doi.org/10.1182/blood-2019-122561

Published: 2019

Journal Articles

Mitapivat (AG-348), an Oral PK-R Activator, in Adults with Non-Transfusion Dependent Thalassemia: A Phase 2, Open-Label, Multicenter Study in Progress

Kevin H. M. Kuo, MDMSc,FRCPC, D. Mark Layton, MB BS, Katrin Uhlig, MD MS, Megan Lynch, MSN, NP, Li Liu, PhD, Elliott P. Vichinsky, MD

Blood (2019) 134 (Supplement_1): 2249.

https://doi.org/10.1182/blood-2019-123113

Published: 2019

Journal Articles

Impulse Oscillometry to Evaluate Lung Function in Young Children Under Five Years with Sickle Cell Disease

Lawson Enokekwa, BS, Tony Ngo, BS, Kathleen McKie, MD, Nnenna R Badamosi, MDMPH

Blood (2019) 134 (Supplement_1): 4853.

https://doi.org/10.1182/blood-2019-121349

Published: 2019

Journal Articles

Hereditary Hemolytic Anemias Other Than Thalassemia: Single Center Experience

Gul Nihal Ozdemir, MD, Mehmet Akif Kilic, MD

Blood (2019) 134 (Supplement_1): 4855.

https://doi.org/10.1182/blood-2019-121702

Published: 2019

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

Less (Fe) is more (Hb) in SCA

Hematopoietic stem cell function in β-thalassemia is impaired and is rescued by targeting the bone marrow niche

Antibodies against the erythroferrone N-terminal domain prevent hepcidin suppression and ameliorate murine thalassemia

Single-cell analysis of bone marrow–derived CD34 + cells from children with sickle cell disease and thalassemia

Heme-regulated eIF2α kinase in erythropoiesis and hemoglobinopathies

Correlation between Changes in Cardiac Iron and Hepatic Iron in Pediatric Patients with Thalassemia Major

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

Cerebral Oxygen Delivery and Metabolic Rate in Chronically Anemic Subjects

2'-O-Methoxyethyl Splice-Switching Oligos to Reverse Splicing from IVS2-745 β-Thalassemia Patient Cells: A Foundation for Potential Therapies

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

The Oxygenscan Provides Clinically Relevant Biomarkers for Treatment Efficacy That Are Associated with Frequency of Vaso-Occlusive Crisis in Sickle Cell Disease

Opioid Analgesics Are Associated with Albuminuria in Adult Patients with Sickle Cell Anemia

A Longitudinal Study in Elderly Patients with Non-Transfusion Dependent Thalassemia (NTDT) Emphasizes the Size of Splenomegaly Predicts Transfusion Requirement Later in Life

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

Factor-Associated Risk Factors of Mild Cognitive Impairment in Thalassemia Patients : Probable Role of FGF21

Effects of Luspatercept on Iron Overload and Impact on Responders to Luspatercept: Results from the BELIEVE Trial

Mitapivat (AG-348), an Oral PK-R Activator, in Adults with Non-Transfusion Dependent Thalassemia: A Phase 2, Open-Label, Multicenter Study in Progress

Impulse Oscillometry to Evaluate Lung Function in Young Children Under Five Years with Sickle Cell Disease

Hereditary Hemolytic Anemias Other Than Thalassemia: Single Center Experience

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