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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

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

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.

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.

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.

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

Published: 2019

Journal Articles

Delayed Diagnosis of Anemia in Late Pregnancy: Consequences at Delivery and Unintended Effects of Third Trimester HIV Screening Policy

Nikil V Moodabagil, MD, Sarah Scheiderich Osmundson, MD, Christian Cepeda, BS Candidate, Colleen Morton, MD

Blood (2019) 134 (Supplement_1): 4675.

https://doi.org/10.1182/blood-2019-124910

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

Blood (2019) 134 (Supplement_1): 4632.

https://doi.org/10.1182/blood-2019-129394

Published: 2019

Journal Articles

Experience with Low Dose Thalidomide in Transfusion Dependent Beta Thalassaemia in a Resource Limited Setting

Pallavi Mehta, MD DM, Neha Yadav, BDS , MBA, Priyanka Soni, MD FNB, Shinto Francis Thekuddan, MBBS,MDDM, Reema Singh, MSc, Vishvdeep Khushoo, MBBS,MDDM, Sumeet Prakash Mirgh, MBBS,MDDM, Narendra Agrawal, MD DM, Rayaz Ahmed, MDDM, Jyotsna Kapoor, MSc, Dinesh Bhurani, MD DM, FRCPA

Blood (2019) 134 (Supplement_1): 963.

https://doi.org/10.1182/blood-2019-131682

Published: 2019

Journal Articles

Interrogating RNA Binding Proteins as Novel Regulators of Fetal Hemoglobin Expression

Aoi Wakabayashi, BS, Jeremy D. Grevet, PhD, Xianjiang Lan, PhD, Malini Sharma, BS, Junwei Shi, PhD, Gerd A. Blobel, MD PhD

Blood (2019) 134 (Supplement_1): 966.

https://doi.org/10.1182/blood-2019-125901

Published: 2019

Journal Articles

Pharmacokinetics of Ticagrelor in Infants and Toddlers Aged <24 Months with Sickle Cell Disease

Baba PD Inusa, MD, Adlette Inati, MD, Philip Maes, MD, Jessie Githanga, MBChB, MMed, Miguel R. Abboud, MD, Maurizio Miano, Elena Cela, Videlis Nduba, Mohammad Niazi, Magnus Åstrand, Kevin Persson, Anders Rikard Berggren, MD PhD, Glenn Carlson, MD

Blood (2019) 134 (Supplement_1): 1005.

https://doi.org/10.1182/blood-2019-129855

Published: 2019

Journal Articles

Selecting ß-Thalassemia Patients for Gene Therapy: A Decision-Making Algorithm

Donatella Baronciani, MD, Maddalena Casale, MD, Lucia De Franceschi, MD, Giovanna Graziadei, MD, Filomena Longo, MD, Raffaella Origa, MD, Valeria Maria Pinto, MD, Paolo Rigano, MD, Monia Marchetti, MD, Antonia Gigante, Emanuele Angelucci, MD, Maria Domenica Cappellini, MD, Achille Iolascon, MD PhD, Antonio Piga, Gian Luca Forni

Blood (2019) 134 (Supplement_1): 972.

https://doi.org/10.1182/blood-2019-128320

Published: 2019

Journal Articles

Characterization of the Severe Phenotype of Pyruvate Kinase Deficiency

Blood (2019) 134 (Supplement_1): 949.

https://doi.org/10.1182/blood-2019-125352

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

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

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

Delayed Diagnosis of Anemia in Late Pregnancy: Consequences at Delivery and Unintended Effects of Third Trimester HIV Screening Policy

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

Experience with Low Dose Thalidomide in Transfusion Dependent Beta Thalassaemia in a Resource Limited Setting

Interrogating RNA Binding Proteins as Novel Regulators of Fetal Hemoglobin Expression

Pharmacokinetics of Ticagrelor in Infants and Toddlers Aged <24 Months with Sickle Cell Disease

Selecting ß-Thalassemia Patients for Gene Therapy: A Decision-Making Algorithm

Characterization of the Severe Phenotype of Pyruvate Kinase Deficiency

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