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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

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

Autism-Associated Chromatin Remodeler CHD8 Governs the Survival and Differentiation of Hematopoietic Stem/Progenitor Cells

Zhaowei Tu, PhD, Chen Wang, MSc, Chuntao Zhao, PhD, Lei Huang, PhD, Nathan Salomonis, PhD, Richard Q Lu, PhD, Yi Zheng, PhD

Blood (2019) 134 (Supplement_1): 1191.

https://doi.org/10.1182/blood-2019-127313

Published: 2019

Journal Articles

Targeting Transcriptional Co-Activator PGC-1a for the Treatment of Sickle Cell Disease

Alawi Habara, MBBS, MSc, Cuong LE, PhD, David H.K. Chui, MD, Martin H. Steinberg, MD, Shuaiying Cui, PhD

Blood (2019) 134 (Supplement_1): 2254.

https://doi.org/10.1182/blood-2019-123959

Published: 2019

Journal Articles

CRISPR-Cas9 Genome Editing of γ-Globin Promoters in Human Hematopoietic Stem Cells to Induce Erythrocyte Fetal Hemoglobin for Treatment of β-Hemoglobinopathies

Blood (2019) 134 (Supplement_1): 2066.

https://doi.org/10.1182/blood-2019-131318

Published: 2019

Journal Articles

Understanding Heterogeneity of Fetal Hemoglobin Induction through Comparative Analysis of Stage-Matched F- and a-Cells

Eugene Khandros, MD PhD, Peng Huang, PhD, Scott A. Peslak, MD PhD, Belinda Giardine, Zhe Zhang, PhD, Cheryl A Keller, PhD, Ross C. Hardison, PhD, Gerd A. Blobel, MD PhD

Blood (2019) 134 (Supplement_1): 981.

https://doi.org/10.1182/blood-2019-124099

Published: 2019

Journal Articles

Bach1 Inhibitors Reduce Stasis in Townes Sickle Cell Mouse Model

John D Belcher, PhD, Margaret Biddle, PhD, Domi Stickens, PhD, Lyndsay Olson, Eric Bell, Chunsheng Chen, Fuad Abdulla, Julia Nguyen, Zachary Monroe Kiser, BS, PhD, Jacob Lloyd, MD, Gregory M Vercellotti, MD

Blood (2019) 134 (Supplement_1): 983.

https://doi.org/10.1182/blood-2019-127300

Published: 2019

Journal Articles

Pharmacokinetics-Guided Dosing of Hydroxyurea Can Achieve Near-Pancellular Fetal Hemoglobin Expression in Sickle Cell Anemia: F-Cell Analysis As a Benchmark for Disease-Modifying Therapy

Charles T. Quinn, MD, Omar Niss, MD, Amanda Pfeiffer, Jennifer Korpik, Holly Bonar, Mary Reynaud, Russell E. Ware, MD PhD, Patrick T. McGann, MD

Blood (2019) 134 (Supplement_1): 892.

https://doi.org/10.1182/blood-2019-130414

Published: 2019

Journal Articles

Single Nucleotide Polymorphisms in SAR1A Codon Regions Associated with Hydroxyurea Response in Sickle Cell Disease and Potentially Influenced in Mirnas Binding

Chutima Kumkhaek, PhD, Christine Kim, James G. Taylor, MD, Jianqiong Zhu, MD, Wulin Aerbajinai, MD, Griffin Rodgers, MD

Blood (2019) 134 (Supplement_1): 987.

https://doi.org/10.1182/blood-2019-124965

Published: 2019

Journal Articles

Chromatin Accessibility Mapping of Primary Erythroid Cell Populations Leads to Identification and Validation of Nuclear Factor I X (NFIX) As a Novel Fetal Hemoglobin (HbF) Repressor

Mudit Chaand, PhD, Chris Fiore, PhD, Brian T Johnston, MS, Diane H Moon, PhD, John P Carulli, PhD, Jeffrey R Shearstone, PhD

Blood (2019) 134 (Supplement_1): 812.

https://doi.org/10.1182/blood-2019-124337

Published: 2019

Journal Articles

The DNA Methylation Maintenance Protein UHRF1 Regulates Fetal Globin Expression Independent of HBG Promoter DNA Methylation

Ruopeng Feng, PhD, Phillip A Doerfler, PhD, Yu Yao, MD, Xing Tang, PhD, Yong-Dong Wang, PhD, Chunliang Li, PhD, David E. Root, PhD, Yong Cheng, PhD, Mitchell J. Weiss, MD PhD

Blood (2018) 132 (Supplement 1): 410.

https://doi.org/10.1182/blood-2018-99-115589

Published: 2018

Journal Articles

Persistence of CRISPR/Cas9-Edited Hematopoietic Stem and Progenitor Cells and Reactivation of Fetal Hemoglobin in Nonhuman Primates

Olivier Humbert, PhD, Stefan Radtke, PhD, Ray R Carillo, Anai M Perez, Sowmya Somashekar Reddy, Lauren E Schefter, Christopher Lux, MDPhD, Sowmya Pattabhi, PhD, Olivier Negre, PhD, Ciaran M Lee, PhD, Jennifer Adair, PhD, Christopher Peterson, PhD, Gang Bao, PhD, David J. Rawlings, MD, Andrew M. Scharenberg, MD, Hans-Peter Kiem, MD PhD

Blood (2018) 132 (Supplement 1): 806.

https://doi.org/10.1182/blood-2018-99-112996

Published: 2018

Journal Articles

Benserazide As a Novel Fetal Hemoglobin Inducer: An Observational Study in Non-Carriers of Hemoglobin Disorders

Marina Erê A. H. P. Santos, BS, Felipe Vendrame, MSc, Alvaro Henrique Junqueira Tavares, BS, Leticia Marani, MSc, Paula C. de Azevedo, MD, Luiza G Piovesana, MD, Fernando F. Costa, MD, PhD, Kleber Yotsumoto Fertrin, MD PhD

Blood (2018) 132 (Supplement 1): 2345.

https://doi.org/10.1182/blood-2018-99-118997

Published: 2018

Journal Articles

Engineered Human Umbilical Cord Derived Erythroid Progenitor Cells (HUDEP2) with Sickle or β-Thalassemia Mutation: An in-Vitro System for Testing Pharmacological Induction of Fetal Hemoglobin

So Hyun Park, BS, Ciaran M Lee, PhD, Yankai Zhang, PhD, Alicia Chang, MD, Vivien A Sheehan, MD PhD, Gang Bao, PhD

Blood (2018) 132 (Supplement 1): 3478.

https://doi.org/10.1182/blood-2018-99-114241

Published: 2018

Journal Articles

The LSD1 Inhibitor RN-1 Increases γ-Globin Expression in Baboons By Targeting an Early Event Responsible for γ-Globin Repression

Kestis Vaitkus, Vinzon Ibanez, B.S., Maria Armila Ruiz, B.S., Ramasamy Jagadeeswaran, PhD, Yogenthiran Saunthararajah, MD, James Douglas Engel, PhD, Joseph DeSimone, PhD, Angela Rivers, MD, Donald Lavelle, PhD

Blood (2018) 132 (Supplement 1): 1054.

https://doi.org/10.1182/blood-2018-99-113539

Published: 2018

Journal Articles

Flipping the Switch: Initial Results of Genetic Targeting of the Fetal to Adult Globin Switch in Sickle Cell Patients

Blood (2018) 132 (Supplement 1): 1023.

https://doi.org/10.1182/blood-2018-99-116733

Published: 2018

Journal Articles

Talen Mediated Therapeutic Gene Editing Strategy for ß-Hemoglobinopathies

Christopher Lux, MDPhD, Kyle Jacoby, PhD, Sowmya Pattabhi, PhD, Julia Yang, BS, Olivier Humbert, PhD, Brian Gordon, MS, Irwin D. Bernstein, MD, David J. Rawlings, MD, Andrew M. Scharenberg, MD

Blood (2017) 130 (Supplement 1): 3340.

https://doi.org/10.1182/blood.V130.Suppl_1.3340.3340

Published: 2017

Journal Articles

Novel Conjugates of Butyrate and δ-Aminolevulinate Increase γ-Globin Gene Expression and Fetal Hemoglobin Synthesis in Erythroid Progenitors

Aluya R. Oseghale, BSc, Marjorie M. Johnson, BS, Xingguo Zhu, PhD, Abraham Nudelman, PhD, Ada Rephaeli, PhD, Betty S. Pace, MD

Blood (2017) 130 (Supplement 1): 3511.

https://doi.org/10.1182/blood.V130.Suppl_1.3511.3511

Published: 2017

Journal Articles

New Insights into F-Cells, F-Reticulocytes and the Role of Fetal Hemoglobin in Protecting Sickle Red Cells from Hemolysis upon Mechanical Stress

Sara El Hoss, Maria Alejandra Lizarralde Iragorri, Valentine Brousse, MD, Michaël Dussiot, PhD, Sophie Lefevre, PhD, Alexander Rodrigo Ferreira, Yann Lamarre, PhD, Ana Cristina Silva Pinto, MD PhD, Simone Kashima, PhD, Claudine Lapoumeroulie, PhD, Dimas Tadeu Covas, MD PhD, Caroline Le Van Kim, PhD, Yves Colin Aronovicz, PhD, Jacques Elion, MD PhD, Olivier Français, PhD, Bruno Le Pioufle, PhD, Wassim El Nemer, PhD

Blood (2017) 130 (Supplement 1): 3513.

https://doi.org/10.1182/blood.V130.Suppl_1.3513.3513

Published: 2017

Journal Articles

Oral Administration of the LSD1 Inhibitor OG-S1335 Increases Fetal Hemoglobin in Humanized Transgenic Sickle Cell Disease Mice and in Baboons

Angela Rivers, MD PhD, Kestis Vaitkus, Ramasamy Jagadeeswaran, PhD MSc, Vinzon Ibanez, B.S., Maria Armila Ruiz, B.S., Filippo Ciceri, PhD, Fernando Cavalcanti, PhD, Robert E. Molokie, MD, Donald Lavelle, PhD

Blood (2017) 130 (Supplement 1): 356.

https://doi.org/10.1182/blood.V130.Suppl_1.356.356

Published: 2017

NARROW

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

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

Autism-Associated Chromatin Remodeler CHD8 Governs the Survival and Differentiation of Hematopoietic Stem/Progenitor Cells

Targeting Transcriptional Co-Activator PGC-1a for the Treatment of Sickle Cell Disease

CRISPR-Cas9 Genome Editing of γ-Globin Promoters in Human Hematopoietic Stem Cells to Induce Erythrocyte Fetal Hemoglobin for Treatment of β-Hemoglobinopathies

Understanding Heterogeneity of Fetal Hemoglobin Induction through Comparative Analysis of Stage-Matched F- and a-Cells

Bach1 Inhibitors Reduce Stasis in Townes Sickle Cell Mouse Model

Pharmacokinetics-Guided Dosing of Hydroxyurea Can Achieve Near-Pancellular Fetal Hemoglobin Expression in Sickle Cell Anemia: F-Cell Analysis As a Benchmark for Disease-Modifying Therapy

Single Nucleotide Polymorphisms in SAR1A Codon Regions Associated with Hydroxyurea Response in Sickle Cell Disease and Potentially Influenced in Mirnas Binding

Chromatin Accessibility Mapping of Primary Erythroid Cell Populations Leads to Identification and Validation of Nuclear Factor I X (NFIX) As a Novel Fetal Hemoglobin (HbF) Repressor

The DNA Methylation Maintenance Protein UHRF1 Regulates Fetal Globin Expression Independent of HBG Promoter DNA Methylation

Persistence of CRISPR/Cas9-Edited Hematopoietic Stem and Progenitor Cells and Reactivation of Fetal Hemoglobin in Nonhuman Primates

Benserazide As a Novel Fetal Hemoglobin Inducer: An Observational Study in Non-Carriers of Hemoglobin Disorders

Engineered Human Umbilical Cord Derived Erythroid Progenitor Cells (HUDEP2) with Sickle or β-Thalassemia Mutation: An in-Vitro System for Testing Pharmacological Induction of Fetal Hemoglobin

The LSD1 Inhibitor RN-1 Increases γ-Globin Expression in Baboons By Targeting an Early Event Responsible for γ-Globin Repression

Flipping the Switch: Initial Results of Genetic Targeting of the Fetal to Adult Globin Switch in Sickle Cell Patients

Talen Mediated Therapeutic Gene Editing Strategy for ß-Hemoglobinopathies

Novel Conjugates of Butyrate and δ-Aminolevulinate Increase γ-Globin Gene Expression and Fetal Hemoglobin Synthesis in Erythroid Progenitors

New Insights into F-Cells, F-Reticulocytes and the Role of Fetal Hemoglobin in Protecting Sickle Red Cells from Hemolysis upon Mechanical Stress

Oral Administration of the LSD1 Inhibitor OG-S1335 Increases Fetal Hemoglobin in Humanized Transgenic Sickle Cell Disease Mice and in Baboons

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American Society of Hematology

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American Society of Hematology

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