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Journal Articles

Fetal Hemoglobin Promotes P. Falciparum Growth in Sickle Cell Disease Erythrocytes

Natasha M. Archer, MD, Manoj Duraisingh

Blood (2019) 134 (Supplement_1): 2252.

https://doi.org/10.1182/blood-2019-130763

Published: 2019

Journal Articles

Identification of Novel Variants Associated with Fetal Hemoglobin Levels in Healthy Donors (the INTERVAL study)

Emily Kawabata, Samuel Lessard, PhD, Dirk Paul, Paola G Bronson, Robert Peters, Sriram Krishnamoorthy, PhD, William Astle, Adam Butterworth

Blood (2019) 134 (Supplement_1): 2243.

https://doi.org/10.1182/blood-2019-123977

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

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

Editing a γ-Globin Repressor Binding Site Restores Fetal Hemoglobin Synthesis and Corrects the Phenotype of Sickle Cell Disease Erythrocytes

Leslie Weber, Giacomo Frati, Tristan Felix, Clara Wollenschlaeger, Antonio Casini, Vasco Meneghini, Cecile Masson, Anne De Cian, Anne Chalumeau, Fulvio Mavilio, PhD, Mario Amendola, Isabelle André, PhD, Anna Cereseto, Wassim El Nemer, PhD, Jean-Paul Concordet, Carine Giovannangeli, Marina Cavazzana, Annarita Miccio, PhD

Blood (2019) 134 (Supplement_1): 4635.

https://doi.org/10.1182/blood-2019-123192

Published: 2019

Journal Articles

EDIT-301: An Experimental Autologous Cell Therapy Comprising Cas12a-RNP Modified mPB-CD34+ Cells for the Potential Treatment of SCD

Edouard De Dreuzy, PhD, Jack Heath, John A Zuris, PhD, Patricia Sousa, Ramya Viswanathan, Sean Scott, Jen Da Silva, Terence Ta, PhD, Stacy Capehart, PhD, Tongyao Wang, Cecilia Fernandez, PhD, Vic E Myer, PhD, Charles F Albright, PhD, Christopher J Wilson, PhD, Sandra Teixeira, PhD, Kai-Hsin Chang, PhD

Blood (2019) 134 (Supplement_1): 4636.

https://doi.org/10.1182/blood-2019-130256

Published: 2019

Journal Articles

High Hemoglobin F in Sickle Cell Disease: Waning Protection with Age

Seda S. Tolu, MD, Morayma Reyes Gil, MD PhD, Ugochi Olivia Ogu, MD, Jui Choudhuri, MD, Roger Fecher, MD, Gracy Sebastian, NP, Caterina P. Minniti, MD

Blood (2019) 134 (Supplement_1): 3576.

https://doi.org/10.1182/blood-2019-122387

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

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

Red Blood Cell Adhesion Is Increased in the Young Adult Population with Sickle Cell Disease

Erina Quinn, BA, Charlotte Yuan, BA, Sargam Kapoor, MD, Karen Ireland, RN, Janine Keenan, RN, Deepa Manwani, MD, Umut A. Gurkan, PhD, Jane Little, MD

Blood (2018) 132 (Supplement 1): 2387.

https://doi.org/10.1182/blood-2018-99-120175

Published: 2018

Journal Articles

A Single -195 C < G HBG1 Promoter Mediated By CRISPR/Cas9 Genome Editing Induces Fetal Hemoglobin Synthesis in Hudep-2

Priscila Keiko Matsumoto Martin, PhD, Dulcinéia Martins de Albuquerque, PhD, Carolina Lanaro, PhD, Ryo Kurita, PhD, Yukio Nakamura, MD PhD, Fernando F. Costa, MD, PhD

Blood (2018) 132 (Supplement 1): 3481.

https://doi.org/10.1182/blood-2018-99-118534

Published: 2018

Journal Articles

Reduced Plasmodium Falciparum Growth in Erythrocytes Is Associated with Fetal Hemoglobin Expression

Natasha M. Archer, MD, Nicole Petersen, Jane S. Hankins, MD MS, Manoj T Duraisingh, PhD

Blood (2018) 132 (Supplement 1): 1067.

https://doi.org/10.1182/blood-2018-99-117857

Published: 2018

Journal Articles

Comparison of Fetal and Adult Erythroid Chromosomal Architectures Identifies a Novel Fetal Hemoglobin Regulatory Region

Peng Huang, PhD, Cheryl A. Keller, PhD, Belinda Giardine, James O.J. Davies, Jim R. Hughes, Ross C Hardison, PhD, Gerd Blobel, MD PhD

Blood (2017) 130 (Supplement 1): 774.

https://doi.org/10.1182/blood.V130.Suppl_1.774.774

Published: 2017

Journal Articles

Low Impact of Genetic Modifiers on the Phenotype of Homozygous Beta Thalassemia in the Last Decennial Cohort of Thalassemia Newborns in Sardinia

Paolo Moi, MD, Matteo Marcello, MD, Stefania Satta, PhD, Laura Manunza, PhD, Franca Rosa De Martis, Raffaella Origa, MD, Giovanni Caocci, MD, Susanna Barella, MD

Blood (2017) 130 (Supplement 1): 951.

https://doi.org/10.1182/blood.V130.Suppl_1.951.951

Published: 2017

Journal Articles

CRISPR/Cas9 Genome Editing to Treat Sickle Cell Disease and B-Thalassemia: Re-Creating Genetic Variants to Upregulate Fetal Hemoglobin Appear Well-Tolerated, Effective and Durable.

Blood (2017) 130 (Supplement 1): 284.

https://doi.org/10.1182/blood.V130.Suppl_1.284.284

Published: 2017

Journal Articles

KLF1 drives the expression of fetal hemoglobin in British HPFH

Beeke Wienert, Gabriella E. Martyn, Ryo Kurita, Yukio Nakamura, Kate G. R. Quinlan, Merlin Crossley

Blood (2017) 130 (6): 803–807.

https://doi.org/10.1182/blood-2017-02-767400

Published: 2017

Includes: Supplemental data

Journal Articles

An HPFH Mutation in the γ-Globin Gene Promoter of Humanized Cooley's Anemia Mice Rescues Them from Perinatal Lethality and Renders Them Transfusion Independent

Suean Daimia Fontenard, BS, Yongliang Huo, PhD, Shanrun Liu, MD PhD, Jonathan Lockhart, BS, Michael Berlett, BS, Thomas M Ryan, PhD

Blood (2016) 128 (22): 1290.

https://doi.org/10.1182/blood.V128.22.1290.1290

Published: 2016

Journal Articles

Induction of Fetal Hemoglobin Synthesis By Crispr/Cas9-Mediated Disruption of the β-Globin Locus Architecture

Chiara Antoniani, MSc, Vasco Meneghini, Annalisa Lattanzi, PhD, Giulia Pavani, PhD, Tristan Felix, MSc, Fatima Amor, MSc, Oriana Romano, Elisa Magrin, PhD, Leslie Weber, MSc, Thomas Cradick, Ante S Lundberg, MD, Matthew Porteus, MD PhD, Mario Amendola, Marina Cavazzana, MD PhD, Fulvio Mavilio, PhD, Annarita Miccio, PhD

Blood (2016) 128 (22): 321.

https://doi.org/10.1182/blood.V128.22.321.321

Published: 2016

Journal Articles

Crispr/Cas9- Mediated Genome Editing of Human CD34 + Cells Upregulate Fetal Hemoglobin to Clinically Relevant Levels in Single Cell-Derived Erythroid Colonies

Bibhu Mishra, PhD, Song Chou, PhD, Michelle I Lin, PhD, Elizabeth J Paik, PhD, Ying Zhang, PhD, Ruibin Liang, MS, Kaleigh Tomkinson, MS, Michael Pettiglio, MS, Andrew Sanginario, BS, Melanie Allen, MS, Thomas Cradick, PhD, Matthias John, PhD, Tirtha Chakraborty, PhD, Chad A Cowan, PhD, Rodger Novak, MD, Ante S Lundberg, MD

Blood (2016) 128 (22): 3623.

https://doi.org/10.1182/blood.V128.22.3623.3623

Published: 2016

NARROW

Fetal Hemoglobin Promotes P. Falciparum Growth in Sickle Cell Disease Erythrocytes

Identification of Novel Variants Associated with Fetal Hemoglobin Levels in Healthy Donors (the INTERVAL study)

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

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

Editing a γ-Globin Repressor Binding Site Restores Fetal Hemoglobin Synthesis and Corrects the Phenotype of Sickle Cell Disease Erythrocytes

EDIT-301: An Experimental Autologous Cell Therapy Comprising Cas12a-RNP Modified mPB-CD34+ Cells for the Potential Treatment of SCD

High Hemoglobin F in Sickle Cell Disease: Waning Protection with Age

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

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

Red Blood Cell Adhesion Is Increased in the Young Adult Population with Sickle Cell Disease

A Single -195 C < G HBG1 Promoter Mediated By CRISPR/Cas9 Genome Editing Induces Fetal Hemoglobin Synthesis in Hudep-2

Reduced Plasmodium Falciparum Growth in Erythrocytes Is Associated with Fetal Hemoglobin Expression

Comparison of Fetal and Adult Erythroid Chromosomal Architectures Identifies a Novel Fetal Hemoglobin Regulatory Region

Low Impact of Genetic Modifiers on the Phenotype of Homozygous Beta Thalassemia in the Last Decennial Cohort of Thalassemia Newborns in Sardinia

CRISPR/Cas9 Genome Editing to Treat Sickle Cell Disease and B-Thalassemia: Re-Creating Genetic Variants to Upregulate Fetal Hemoglobin Appear Well-Tolerated, Effective and Durable.

KLF1 drives the expression of fetal hemoglobin in British HPFH

An HPFH Mutation in the γ-Globin Gene Promoter of Humanized Cooley's Anemia Mice Rescues Them from Perinatal Lethality and Renders Them Transfusion Independent

Induction of Fetal Hemoglobin Synthesis By Crispr/Cas9-Mediated Disruption of the β-Globin Locus Architecture

Crispr/Cas9- Mediated Genome Editing of Human CD34 + Cells Upregulate Fetal Hemoglobin to Clinically Relevant Levels in Single Cell-Derived Erythroid Colonies

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American Society of Hematology

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American Society of Hematology

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