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Journal Articles

Therapeutic Base Editing of Human Hematopoietic Stem Cells

Jing Zeng, MD, Yuxuan Wu, PhD, Chunyan Ren, PhD, Jasmine Bonanno, Anne H. Shen, Devlin Shea, Jason M Gehrke, PhD, John P Manis, MD, J. Keith Gehrke, Daniel E. Bauer, MD PhD

Blood (2019) 134 (Supplement_1): 612.

https://doi.org/10.1182/blood-2019-129173

Published: 2019

Journal Articles

Codanin-1 Mutations Engineered in Human Erythroid Cells Allow Investigation of Mechanisms Underlying Congenital Dyserythropoietic Anemia

Zachary C. Murphy, PhD, Michael Getman, MS, Jacquelyn Lillis, Laurie A. Steiner, MD

Blood (2019) 134 (Supplement_1): 3739.

https://doi.org/10.1182/blood-2019-127415

Published: 2019

Journal Articles

Whole Genome Sequencing Identifies Small Deletions in Ribosomal Genes Causing Diamond Blackfan Anemia

David M. Bodine, PhD, Jens Lichtenberg, Nancy Seidel, NISC Comparative SequencingProgram, G. Jayashree Jagadeesh, John G Conboy, PhD, Jason E. Farrar, MD, Jessica Kang, BS, Lionel Blanc, PhD, Evangelia Atsidaftos, MA, MPH, Steven Ellis, PhD, Jeffrey Michael M Lipton, MD PhD, Adrianna Vlachos, MD

Blood (2019) 134 (Supplement_1): 2502.

https://doi.org/10.1182/blood-2019-121710

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

Knockdown of HNF4A Gene Increases Fetal Hemoglobin Synthesis in Hudep-2

Ingrid Grazielle Sousa, BSc, MSc, Priscila Keiko Matsumoto Martin, PhD, Dulcinéia Martins de Albuquerque, BSc, PhD, Carolina Lanaro, BSc, PhD, Ryo Kurita, PhD, Yukio Nakamura, MD PhD, Fernando Ferreira Costa, MD PhD

Blood (2019) 134 (Supplement_1): 968.

https://doi.org/10.1182/blood-2019-130920

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

Blood (2019) 134 (Supplement_1): 3578.

https://doi.org/10.1182/blood-2019-130758

Published: 2019

Journal Articles

Common Myeloid Progenitors As Biomarkers of Hbf Response to Hydroxyurea in Sickle Cell Disease

Caterina P. Minniti, MD, Seda S. Tolu, MD, Kai Wang, PhD, Zi Yan, Andrew Crouch, Gracy Sebastian, NP, Mark Chaitowitz, MD, Evan Schwechter, MD, Joan Uehlinger, MD, Deepa Manwani, MD, Eric Fornari, MD, Eric E. Bouhassira, PhD

Blood (2019) 134 (Supplement_1): 4827.

https://doi.org/10.1182/blood-2019-131466

Published: 2019

Journal Articles

KLF4 Is Needed for Optimal Hydroxyurea Induction of Gamma-Globin in K562 Cells

Alireza Paikari, MD, Ankush Goyal, MA, Celeste K. Kanne, BA, Vivien A. Sheehan, MD PhD

Blood (2019) 134 (Supplement_1): 971.

https://doi.org/10.1182/blood-2019-131214

Published: 2019

Journal Articles

Genetic Analysis in the Tanzania Sickle Surveillance Study (TS3): Modifiers of Sickle Cell Disease and Identification of Hemoglobin Variants

Luke R. Smart, MD, Emmanuela E. Ambrose, MD, Mwesige Charles, BScHLS, Arielle G. Hernandez, MPH, Teresa S. Latham, MA, Adolfine Hokororo, MD MMed, MSc, Medard Beyanga, BLS, MSc, Erasmus Kamugisha, MD MSc, PhD, Erius Tebuka, MD MMed, Thad A. Howard, MS, Russell E. Ware, MD PhD

Blood (2019) 134 (Supplement_1): 988.

https://doi.org/10.1182/blood-2019-128554

Published: 2019

Journal Articles

Regulation of Fetal Hemoglobin through the Insulin Signaling Pathway

Alireza Paikari, MD, Yankai Zhang, PhD, Alicia Chang, MD, Ankush Goyal, MA, Evadnie Rampersaud, PhD, Jonathan M. Flanagan, PhD, Mitchell J. Weiss, MD, PhD, Vivien A. Sheehan, MD PhD

Blood (2019) 134 (Supplement_1): 811.

https://doi.org/10.1182/blood-2019-131313

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

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

Blood (2019) 134 (Supplement_1): 3352.

https://doi.org/10.1182/blood-2019-126710

Published: 2019

Journal Articles

The Role of Adenosine Monophosphate Activated Protein Kinase Alpha 1 (AMPK) in Gamma-Globin Regulation

Alicia Chang, MD, Yankai Zhang, PhD, Nelda Itzep, MD, Vivien A Sheehan, MD PhD

Blood (2018) 132 (Supplement 1): 413.

https://doi.org/10.1182/blood-2018-99-117092

Published: 2018

Journal Articles

Insulin-like Growth Factor Binding Protein-3 (IGFBP3) Induces Fetal Hemoglobin in Hematopoietic Stem and Progenitor Cells from Patients with Sickle Cell Anemia

Alireza Paikari, MD, Tian Mi, PhD, Yankai Zhang, PhD, Evadnie Rampersaud, PhD, Guolian Kang, PhD, Gang Wu, PhD, Jane S. Hankins, MD MS, Jeremie H. Estepp, MD, Pavel Sumazin, PhD, Mitchell J. Weiss, MD PhD, Vivien A Sheehan, MD PhD

Blood (2018) 132 (Supplement 1): 722.

https://doi.org/10.1182/blood-2018-99-115383

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

Comparative Studies Reveal Robust HbF Induction By Editing of HBG1/2 Promoters or BCL11A Erythroid-Enhancer in Human CD34+ Cells but That BCL11A Erythroid-Enhancer Editing Is Associated with Selective Reduction in Erythroid Lineage Reconstitution in a Xenotransplantation Model

Blood (2018) 132 (Supplement 1): 409.

https://doi.org/10.1182/blood-2018-99-114780

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

Structural Variation within the Beta-Globin Gene Cluster Among HbS Haplotype Groups

Pamela Lurie, MS, Bamidele Tayo, PhD, Richard S. Cooper, MD, Guillame Lettre, PhD, Ambroise Wonkman, MD PhD, George Ayodo, Stephen K. Obaro, MD PhD, Titilola S. Akingbola, MBBS, FWACP, Neil Hanchard, MD PhD

Blood (2018) 132 (Supplement 1): 2365.

https://doi.org/10.1182/blood-2018-99-119824

Published: 2018

Journal Articles

Gene Therapy for Sickle Cell Anemia Using a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant Shows Promising Correction of the Disease Phenotype

Blood (2018) 132 (Supplement 1): 1021.

https://doi.org/10.1182/blood-2018-99-119591

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

NARROW

Therapeutic Base Editing of Human Hematopoietic Stem Cells

Codanin-1 Mutations Engineered in Human Erythroid Cells Allow Investigation of Mechanisms Underlying Congenital Dyserythropoietic Anemia

Whole Genome Sequencing Identifies Small Deletions in Ribosomal Genes Causing Diamond Blackfan Anemia

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

Knockdown of HNF4A Gene Increases Fetal Hemoglobin Synthesis in Hudep-2

Establishing a Link between Zinc Homeostasis and Globin Gene Expression

Common Myeloid Progenitors As Biomarkers of Hbf Response to Hydroxyurea in Sickle Cell Disease

KLF4 Is Needed for Optimal Hydroxyurea Induction of Gamma-Globin in K562 Cells

Genetic Analysis in the Tanzania Sickle Surveillance Study (TS3): Modifiers of Sickle Cell Disease and Identification of Hemoglobin Variants

Regulation of Fetal Hemoglobin through the Insulin Signaling Pathway

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

Complementary Base Editing Approaches for the Treatment of Sickle Cell Disease and Beta Thalassemia

The Role of Adenosine Monophosphate Activated Protein Kinase Alpha 1 (AMPK) in Gamma-Globin Regulation

Insulin-like Growth Factor Binding Protein-3 (IGFBP3) Induces Fetal Hemoglobin in Hematopoietic Stem and Progenitor Cells from Patients with Sickle Cell Anemia

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

Comparative Studies Reveal Robust HbF Induction By Editing of HBG1/2 Promoters or BCL11A Erythroid-Enhancer in Human CD34+ Cells but That BCL11A Erythroid-Enhancer Editing Is Associated with Selective Reduction in Erythroid Lineage Reconstitution in a Xenotransplantation Model

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

Structural Variation within the Beta-Globin Gene Cluster Among HbS Haplotype Groups

Gene Therapy for Sickle Cell Anemia Using a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant Shows Promising Correction of the Disease Phenotype

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

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American Society of Hematology

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American Society of Hematology

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