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Journal Articles

Gene therapy for sickle cell disease: moving from the bench to the bedside

Allistair A. Abraham, John F. Tisdale

Blood (2021) 138 (11): 932–941.

https://doi.org/10.1182/blood.2019003776

Published: 2021

Journal Articles

Badat M, Davies JOJ, Fisher CA, et al. A remarkable case of HbH disease illustrates the relative contributions of the α-globin enhancers to gene expression. Blood . 2021;137(4):572-575.

Blood (2021) 137 (20): 2854.

https://doi.org/10.1182/blood.2021011963

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

A remarkable case of HbH disease illustrates the relative contributions of the α-globin enhancers to gene expression

Mohsin Badat, James O. J. Davies, Christopher A. Fisher, Damien J. Downes, Anna Rose, Andreas B. Glenthøj, Eduard J. van Beers, Cornelis L. Harteveld, Douglas R. Higgs

Blood (2021) 137 (4): 572–575.

https://doi.org/10.1182/blood.2020006680

Published: 2021

Includes: Supplemental data

Journal Articles

Diamond-Blackfan anemia

Lydie Da Costa, Thierry Leblanc, Narla Mohandas

Blood (2020) 136 (11): 1262–1273.

https://doi.org/10.1182/blood.2019000947

Published: 2020

Journal Articles

An evolutionarily ancient mechanism for regulation of hemoglobin expression in vertebrate red cells

Masato Miyata, Nynke Gillemans, Dorit Hockman, Jeroen A. A. Demmers, Jan-Fang Cheng, Jun Hou, Matti Salminen, Christopher A. Fisher, Stephen Taylor, Richard J. Gibbons, Jared J. Ganis, Leonard I. Zon, Frank Grosveld, Eskeatnaf Mulugeta, Tatjana Sauka-Spengler, Douglas R. Higgs, Sjaak Philipsen

Blood (2020) 136 (3): 269–278.

https://doi.org/10.1182/blood.2020004826

Published: 2020

Includes: Multimedia, Supplemental data

Journal Articles

A Cambrian origin for globin gene regulation

Ross C. Hardison

Blood (2020) 136 (3): 261–262.

https://doi.org/10.1182/blood.2020006649

Published: 2020

Journal Articles

A master erythroid regulator gets its own GPS

Lily Jun-shen Huang, James J. Bieker

Blood (2020) 135 (25): 2209–2210.

https://doi.org/10.1182/blood.2020005882

Published: 2020

Journal Articles

The HRI-regulated transcription factor ATF4 activates BCL11A transcription to silence fetal hemoglobin expression

Peng Huang, Scott A. Peslak, Xianjiang Lan, Eugene Khandros, Jennifer A. Yano, Malini Sharma, Cheryl A. Keller, Belinda Giardine, Kunhua Qin, Osheiza Abdulmalik, Ross C. Hardison, Junwei Shi, Gerd A. Blobel

Blood (2020) 135 (24): 2121–2132.

https://doi.org/10.1182/blood.2020005301

Published: 2020

Includes: Supplemental data

Journal Articles

Understanding heterogeneity of fetal hemoglobin induction through comparative analysis of F and A erythroblasts

Eugene Khandros, Peng Huang, Scott A. Peslak, Malini Sharma, Osheiza Abdulmalik, Belinda M. Giardine, Zhe Zhang, Cheryl A. Keller, Ross C. Hardison, Gerd A. Blobel

Blood (2020) 135 (22): 1957–1968.

https://doi.org/10.1182/blood.2020005058

Published: 2020

Includes: Supplemental data

Journal Articles

Variable cells with identical genetic codes

C. Babbs, D. R. Higgs

Blood (2020) 135 (22): 1921–1922.

https://doi.org/10.1182/blood.2020005875

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

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

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

O-Glcnacylation Is Essential for Erythropoiesis

Matthew P Parker, BS, Halyna Fedosyuk, MS, Lesya V Novikova, PhD, Zhen Zhang, PhD, Chad Slawson, PhD, Kenneth R Peterson, PhD

Blood (2019) 134 (Supplement_1): 2218.

https://doi.org/10.1182/blood-2019-129468

Published: 2019

Journal Articles

Establishment of a Spontaneous Stromal Microenvironment from Cord Blood Supports Human Dynamic Erythropoietic Temporal Maturation in Long-Term Serum- and Cytokine-Free 3D Cultures and Reveals a Distinct CD44 hi Population

Susana Brito Dos Santos, MSc PhD, Athanasios Mantalaris, PhD, Nicki Panoskaltsis, MD PhD

Blood (2019) 134 (Supplement_1): 2219.

https://doi.org/10.1182/blood-2019-131085

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

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

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Gene therapy for sickle cell disease: moving from the bench to the bedside

Badat M, Davies JOJ, Fisher CA, et al. A remarkable case of HbH disease illustrates the relative contributions of the α-globin enhancers to gene expression. Blood . 2021;137(4):572-575.

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

A remarkable case of HbH disease illustrates the relative contributions of the α-globin enhancers to gene expression

Diamond-Blackfan anemia

An evolutionarily ancient mechanism for regulation of hemoglobin expression in vertebrate red cells

A Cambrian origin for globin gene regulation

A master erythroid regulator gets its own GPS

The HRI-regulated transcription factor ATF4 activates BCL11A transcription to silence fetal hemoglobin expression

Understanding heterogeneity of fetal hemoglobin induction through comparative analysis of F and A erythroblasts

Variable cells with identical genetic codes

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

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

Combining HPFH Mutations in Human Adult HSCs to Enhance Reactivation of Fetal Hemoglobin

O-Glcnacylation Is Essential for Erythropoiesis

Establishment of a Spontaneous Stromal Microenvironment from Cord Blood Supports Human Dynamic Erythropoietic Temporal Maturation in Long-Term Serum- and Cytokine-Free 3D Cultures and Reveals a Distinct CD44 hi Population

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

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

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American Society of Hematology

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