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

Jean-Yves Metais, PhD, Phillip A Doerfler, PhD, Thiyagaraj Mayuranathan, Daniel E. Bauer, MD PhD, Stephanie Fowler, Matthew Hsieh, MD, Varun Katta, Sagar Keriwala, Cicera Lazzarotto, Kevin Luk, Michael Neel, Scott Perry, Shaina Porter, Byoung Y. Ryu, PhD, Akshay Sharma, MBBS, Devlin Shea, Samuel Peters, John F. Tisdale, MD, Scot A. Wolfe, PhD, Kaitly Jensen Woodard, BS, Naoya Uchida, MD, Yuxuan Wu, PhD, Yu Yao, MD, Jing Zeng, MD, Shondra M. Pruett-Miller, PhD, Shengdar Q. Tsai, PhD, Mitchell J. Weiss, MD, PhD
Journal: Blood
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
Journal: Blood
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
Journal: Blood
Blood (2019) 134 (Supplement_1): 4636.
https://doi.org/10.1182/blood-2019-130256
Published: 2019
Journal Articles

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

Natasha M. Archer, MD, Manoj Duraisingh
Journal: Blood
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
Journal: Blood
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
Journal: Blood
Blood (2019) 134 (Supplement_1): 2246.
https://doi.org/10.1182/blood-2019-131487
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
Journal: Blood
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
Journal: Blood
Blood (2019) 134 (Supplement_1): 3352.
https://doi.org/10.1182/blood-2019-126710
Published: 2019
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
Journal: Blood
Blood (2018) 132 (Supplement 1): 3481.
https://doi.org/10.1182/blood-2018-99-118534
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
Journal: Blood
Blood (2018) 132 (Supplement 1): 2387.
https://doi.org/10.1182/blood-2018-99-120175
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
Journal: Blood
Blood (2018) 132 (Supplement 1): 1067.
https://doi.org/10.1182/blood-2018-99-117857
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
Journal: Blood
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

Kai-Hsin Chang, PhD, Minerva Sanchez, Jack Heath, Edouard deDreuzy, PhD, Scott Haskett, MS, Abigail Vogelaar, Kiran Gogi, Jen Da Silva, Tongyao Wang, Andrew Sadowski, Gregory Gotta, Jamaica Siwak, Ramya Viswanathan, Katherine Loveluck, Hoson Chao, Eric Tillotson, Aditi Chalishazar, MS, Abhishek Dass, Frederick Ta, Emily Brennan, Diana Tabbaa, Eugenio Marco, PhD, John A Zuris, PhD, Deepak Reyon, PhD, Meltem Isik, PhD, Ari Friedland, PhD, Terence Ta, PhD, Fred Harbinski, Georgia Giannoukos, PhD, Sandra Teixeira, PhD, Christopher J Wilson, PhD, Charlie Albright, PhD, Haiyan Jiang, PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 409.
https://doi.org/10.1182/blood-2018-99-114780
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
Journal: Blood
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
Journal: Blood
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.

Michelle I. Lin, PhD, Elizabeth Paik, PhD, Bibhu Mishra, PhD, David Burkhardt, Andrew Kernytsky, PhD, Michael Pettiglio, Yi-Shan Chen, Kaleigh Tomkinson, Amanda Woo, Mauricio Cortes, PhD, Siyuan Tan, PhD, Todd D Borland, BS, Lawrence Klein, PhD, Angela Yen, PhD, Sudipta Mahajan, Eric Chan, PhD, Brenda Eustace, PhD, Matthew Porteus, MD PhD, Tirtha Chakraborty, PhD, Chad Cowan, Rodger Novak, MD, Ante Lundberg, MD
Journal: Blood
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

Brief Report
Beeke Wienert, Gabriella E. Martyn, Ryo Kurita, Yukio Nakamura, Kate G. R. Quinlan, Merlin Crossley
Journal: Blood
Blood (2017) 130 (6): 803–807.
https://doi.org/10.1182/blood-2017-02-767400
Published: 2017
Includes: Supplemental data
Journal Articles

Re-Creating Hereditary Persistence of Fetal Hemoglobin (HPFH) to Treat Sickle Cell Disease (SCD) and β-Thalassemia

Michelle I Lin, PhD, Elizabeth J Paik, PhD, Bibhu P Mishra, PhD, Song Chou, PhD, Ying Zhang, PhD, Kaleigh Tomkinson, MS, Michael A Pettiglio, BS, Andrew Sanginario, BS, Amanda Woo, BS, Min Jin Lee, BS, Melanie Allen, MS, Thomas J Cradick, PhD, Tirtha Chakraborty, PhD, Matthew Porteus, MD PhD, Ciaran M Lee, PhD, Gang Bao, PhD, Annarita Miccio, PhD, Annalisa Lattanzi, PhD, Fulvio Mavilio, PhD, Chad A Cowan, PhD, Rodger Novak, MD, Ante S Lundberg, MD
Journal: Blood
Blood (2016) 128 (22): 4708.
https://doi.org/10.1182/blood.V128.22.4708.4708
Published: 2016
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
Journal: Blood
Blood (2016) 128 (22): 1290.
https://doi.org/10.1182/blood.V128.22.1290.1290
Published: 2016
Journal Articles

Nrf2 Activation and Fetal Hemoglobin Induction in Sickle Cell Disease

Sriram Krishnamoorthy, Dipti Gupta, William E. Hobbs, MD PhD, Christine Loh, PhD, David R. Light, Robert Peters, PhD, Sarah Sturtevant, Betty Pace, MD, Yukio Nakamura, Brian Lucas, Benjamin F. Vieira
Journal: Blood
Blood (2016) 128 (22): 4840.
https://doi.org/10.1182/blood.V128.22.4840.4840
Published: 2016