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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
Journal: Blood
Blood (2019) 134 (Supplement_1): 612.
https://doi.org/10.1182/blood-2019-129173
Published: 2019
Journal Articles

Development of a Severity Score System for Thalassemia Syndromes

Angela Vitrano, PhD, Antonella Meloni, PhD, Walter Addario Pollina, Mehran Karimi, MD, Amal El-Beshlawy, MD, Mahmoud Hajipour, Vito Di Marco, Saqib Hussain Ansari, PhD, Aldo Filosa, Paolo Ricchi, MD PhD, Adriana Ceci, MD, Shahina Daar, MD, Sylvia Titi Titi Singer, MD, J F Borgio, MD, Alessia Pepe, MD, Salvatore Scondotto, Gabriella Dardanoni, Rosario Di Maggio, MD, Massimiliano Sacco, DR, Elliott P. Vichinsky, MD, Aurelio Maggio, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2225.
https://doi.org/10.1182/blood-2019-124675
Published: 2019
Journal Articles

Effects of Voxelotor (GBT440) Therapy on Laboratory Testing of Sickle Cell Patients

Michelle E. Savedra, MT(ASCP), Jessica L. Szuberski, MLS ASCP, Tavanna R. Porter, MS, LCGC, Molly S. Hein, Phuong L. Nguyen, MD, Min Shi, MD PhD, Aruna Rangan, MD, James D. Hoyer, MD, Jennifer L. Oliveira, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1011.
https://doi.org/10.1182/blood-2019-131125
Published: 2019
Journal Articles

Molecular Characterization and Frequencies of Different Genetic Ameliorating Factors in Transfusion Dependent Thalassemia Patients from District Peshawar

Gulrukh Mehmood, MBBS, M.Phil, Abid Sohail Taj, PhD MBBS, MRCPath, Arshi Naz, Tariq Masood Khan, PhD MBBS
Journal: Blood
Blood (2019) 134 (Supplement_1): 4816.
https://doi.org/10.1182/blood-2019-130049
Published: 2019
Journal Articles

Genetic Engineering in Hematopoietic Stem Cells for Gene Therapy of Hemoglobinopathies

Giuliana Ferrari, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): SCI-23.
https://doi.org/10.1182/blood-2019-121119
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

Valentina Ghiaccio, PhD, Alisa Dong, PhD, Irene Motta, MD, Shuling Guo, PhD, Raechel Peralta, MS, Susan M Freier, Andy Watt, Yasuhiro Ikawa, MD PhD, Maxwell Chappell, MS, Coralea Stephanou, PhD, Paola Delbini, PhD, Connie Chen, Soteroula Christou, MD, Marina Kleanthous, PhD, Kim Smith-Whitley, MD, Deepa Manwani, MD, Carla Casu, PhD, Maria Domenica Cappellini, MD, Osheiza Abdulmalik, DVM, Stefano Rivella, PhD, Laura Breda, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2244.
https://doi.org/10.1182/blood-2019-127017
Published: 2019
Journal Articles

Durable and Robust Fetal Globin Induction without Anemia in Rhesus Monkeys Following Autologous Hematopoietic Stem Cell Transplant with BCL11A Erythroid Enhancer Editing

Selami Demirci, PhD, Jing Zeng, MD, Yuxuan Wu, PhD, Naoya Uchida, MD, Jackson Gamer, Morgan Yapundich, Claire Drysdale, Aylin C. Bonifacino, Allen E. Krouse, Nathaniel S. Linde, Robert E. Donahue, VMD, Anne H. Shen, Juan J. Haro-Mora, PhD, Alexis Leonard, MD, Tina Nassehi, Kevin Luk, Scot A. Wolfe, Cicera R. Lazzarotto, Shengdar Q. Tsai, PhD, Mitchell J. Weiss, MD, PhD, Daniel E. Bauer, MD PhD, John F. Tisdale, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4632.
https://doi.org/10.1182/blood-2019-129394
Published: 2019
Journal Articles

Activation of ULK1 Kinase Mediates Clearance of Free Alpha-Globin in Human Beta-Thalassemic Erythroblasts

Christophe Lechauve, PhD, Julia Keith, Eugene Khandros, MD PhD, Stephanie Fowler, Kalin Mayberry, Abdullah Freiwan, Christopher Thom, MD PhD, Jingjing Zhang, Heather Tillman, DVM, PhD, Mondira Kundu, MD PhD, Irene Motta, MD, Maria Domenica Cappellini, MD, Mitchell J. Weiss, MD PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 411.
https://doi.org/10.1182/blood-2018-99-113901
Published: 2018
Journal Articles

Highly Efficient Therapeutic Gene Editing of BCL11A enhancer in Human Hematopoietic Stem Cells from ß-Hemoglobinopathy Patients for Fetal Hemoglobin Induction

Jing Zeng, MD, Daniel E. Bauer, MD PhD, Yuxuan Wu, PhD, Benjamin P Roscoe, Pengpeng Liu, Qiuming Yao, PhD, Cicera Lazzarrotto, M. Kendell Clement, Mitchel A. Cole, BS, Kevin Luk, Cristina Baricordi, Erica B. Esrick, MD, John P Manis, MD, David Dorfma, David A. Williams, MD, Alessandra Biffi, MD PhD, Carlo Brugnara, MD, Luca Biasco, Christian Brendel, PhD, Luca Pinello, PhD, Shengdar Tsai, PhD, Scot A. Wolfe
Journal: Blood
Blood (2018) 132 (Supplement 1): 3482.
https://doi.org/10.1182/blood-2018-99-119365
Published: 2018
Journal Articles

A Novel α-Thalassemia Screening Scheme in Neonates with Higher Sensitivity and Specificity Using a Combination of Hb Bart's Quantity and Red Blood Cell Indices

Thidarat Suksangpleng, PhD, Suchada Riolueang, MSc, Jiraporn Korchuenjit, BSc, Supachai Ekwattanakit, PhD MD, Vip Viprakasit, MD DPhil
Journal: Blood
Blood (2018) 132 (Supplement 1): 1059.
https://doi.org/10.1182/blood-2018-99-118216
Published: 2018
Journal Articles

LC-MS Analysis of Anti-Sickling Compounds in Cord Blood Derived RBCs Demonstrates Modification of Fetal Hemoglobin and Globin Chain Binding Preferences

Benjamin Vieira, Vu P. Hong, Kunal Desai, Martin K. Safo, David R. Light
Journal: Blood
Blood (2018) 132 (Supplement 1): 1074.
https://doi.org/10.1182/blood-2018-99-116319
Published: 2018
Journal Articles

The Effect of Ketamine Administration on Pain Control in Painful Crisis of Sickle Cell Anemia Patients during Childhood: A Retrospective Observational Study

Yurdanur Kilinc, MD, Hacer Temizturk, Hayri levent Yılmaz, Metin Cil, Goksel Leblebisatan, Hatice Ilgen Sasmaz, MD
Journal: Blood
Blood (2018) 132 (Supplement 1): 4919.
https://doi.org/10.1182/blood-2018-99-116159
Published: 2018
Journal Articles

Familial Polycythemia Likely Due to Novel Hemoglobin Variant Hemoglobin Hyden

Sandhya Kolagatla, Nagabhishek Moka, MD, Samuel D. Bailey, MD
Journal: Blood
Blood (2018) 132 (Supplement 1): 4925.
https://doi.org/10.1182/blood-2018-99-119590
Published: 2018
Journal Articles

AMPK Regulates the Expression of the Fe-S Cluster Assembly Enzyme (ISCU) and ALAS2, Modulating Cellular Iron Metabolism and Increasing Hemoglobin Synthesis

Ping La, Silvia Pires Lourenco, MSc, Laura Breda, PhD, Stefano Rivella
Journal: Blood
Blood (2018) 132 (Supplement 1): 851.
https://doi.org/10.1182/blood-2018-99-112175
Published: 2018
Journal Articles

Phase 2 Clinical Trial Results for Bitopertin, an Oral Glycine Transporter 1 Inhibitor, in Patients with Non-Transfusion Dependent Beta-Thalassemia

Ali Taher, MD PhD, Vip Viprakasit, MD DPhil, Maria Domenica Cappellini, MD, Dominik Kraus, PhD, Patrick Cech, PhD, Volz Dietmar, PhD, Erica Winter, PharmD, Norman Alan Mazer, MD PhD, Stephane Nave, MD, Juergen Dukart, PhD, Franziska Schaedeli Stark, PhD, Annette Koerner, PhD, Omar Khwaja, MD PhD, Ricardo Hermosilla, DVM, PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 3635.
https://doi.org/10.1182/blood-2018-99-114776
Published: 2018
Journal Articles

Ulk1 Kinase Mediates Clearance of Free α-Globin in β-Thalassemia

Christophe Lechauve, PhD, Eugene Khandros, MD PhD, Christopher Thom, MD PhD, Abdullah Freiwan, Stephanie Fowler, Kalin Mayberry, Julia Keith, Heather Tillman, DVM, PhD, Mondira Kundu, MD PhD, Mitchell J. Weiss, MD PhD
Journal: Blood
Blood (2017) 130 (Supplement 1): 285.
https://doi.org/10.1182/blood.V130.Suppl_1.285.285
Published: 2017
Journal Articles

Domain-Focused CRISPR-Cas9 Screen Identifies Hri Kinase As a Regulator of Fetal Hemoglobin in Adult Human Erythroid Cells

Jeremy D. Grevet, Nicole Hamagami, Xianjiang Lan, PhD, Laavanya Sankaranarayanan, MS, Saurabh Bhardwaj, PhD, Carolyne Face, David Posocco, Osheiza Abdulmalik, DVM, Cheryl A. Keller, PhD, Belinda Giardine, Simone Sidoli, PhD, Ben Garcia, PhD, Stella T Chou, MD, Ross C Hardison, PhD, Chris Vakoc, MD PhD, Junwei Shi, PhD, Gerd A. Blobel, MD PhD
Journal: Blood
Blood (2017) 130 (Supplement 1): 536.
https://doi.org/10.1182/blood.V130.Suppl_1.536.536
Published: 2017
Journal Articles

Genome Editing Strategies to Treat Beta-Hemoglobinopathies

Mitchell J Weiss, MD PhD
Journal: Blood
Blood (2017) 130 (Supplement 1): SCI-16.
https://doi.org/10.1182/blood.V130.Suppl_1.SCI-16.SCI-16
Published: 2017
Journal Articles

Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown, β-Globin Induction and Simultaneous Repression of β-Globin for the Potential Treatment of Sickle Cell Disease

Olivier Negre, PhD, Christian Brendel, PhD, Geoffrey B. Parsons, Min Luo, PhD, Myriam A Armant, PhD, Swaroopa Guda, PhD, Lauryn Christiansen, Gretchen Lewis, BMBS, Gabor Veres, PhD, Melissa Bonner, PhD, David A. Williams, MD
Journal: Blood
Blood (2017) 130 (Supplement 1): 3557.
https://doi.org/10.1182/blood.V130.Suppl_1.3557.3557
Published: 2017
Journal Articles

Gene Expression Profiling of Reticulocytes in Patients with Hereditary Spherocytosis after Depleting Globin Gene Transcripts

Reena Das, MD, Aggarwal Anu, MSc, Manu Jamwal, MSc, Prashant Sharma, MD DNB, DM, Man Updesh Singh Sachdeva, MD, Deepak Bansal, MD, Pankaj Malhotra, MD MBBS
Journal: Blood
Blood (2016) 128 (22): 1255.
https://doi.org/10.1182/blood.V128.22.1255.1255
Published: 2016