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Journal Articles

Introduction to a review series on gene therapy and gene editing for sickle cell disease and hemophilia

Catherine M. Bollard

Blood (2021) 138 (11): 913.

https://doi.org/10.1182/blood.2021012891

Published: 2021

Journal Articles

Pediatric hemophagocytic lymphohistiocytosis

Scott W. Canna, Rebecca A. Marsh

Blood (2020) 135 (16): 1332–1343.

https://doi.org/10.1182/blood.2019000936

Published: 2020

Journal Articles

Hemolysis in the Spleen Drives Erythrocyte Turnover

Thomas Klei, Robin Van Bruggen, PhD, Jill Dalimot, Martijn Veldthuis, Erik Mul, Timo Rademakers, Mark Hoogenboezem, Sietse Quirijn Nagelkerke, Wilfred Van Ijcken, Soren K. Moestrup, MD PhD, Floris van Alphen, Sander Meijer, PhD, Taco Kuijpers, Rob van Zwieten, Pia Svendsen

Blood (2019) 134 (Supplement_1): 946.

https://doi.org/10.1182/blood-2019-124342

Published: 2019

Journal Articles

Successful Bone Marrow Transplantation Using an Immunomyelosuppressive Conditioning in Patients with Severe Congenital Neutropenia: The Results of a Single-Institute

Risa Matsumura, Shiho Nishimura, MD, Yoko Mizoguchi, MD PhD, Mizuka Miki, MD, Maki Taniguchi, MD, Maiko Shimomura, MD PhD, Shuhei Karakawa, MD PhD, Shinji Mochizuki, MD PhD, Takehiko Doi, MD PhD, Hiroshi Kawaguchi, MD PhD, Masao Kobayashi, MD PhD

Blood (2019) 134 (Supplement_1): 1035.

https://doi.org/10.1182/blood-2019-126653

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

Social Vulnerability Is Associated with Emergency Department Dependency in Pediatric Sickle Cell Disease Patients

Kristina Lai, MPH, Peter A. Lane, MD

Blood (2019) 134 (Supplement_1): 4680.

https://doi.org/10.1182/blood-2019-125351

Published: 2019

Journal Articles

A New Step in Understanding of Fanconi Patients Peripheral Stem Cell Harvesting, a Bridge to Gene Therapy

Jean-Sebastien Diana, MD, Sandra Manceau, PhD, Thierry Leblanc, MD, Chloé Couzin, MD, Alessandra Magnani, MD PhD, Elisa Magrin, PhD, Matthieux Bendavid, MD, Laure Joseph, MD, Marianne Delville, MD, Stephane Blanche, MD, Jean Soulier, MD PhD, Marina Cavazzana, MD PhD, Francois Lefrere, MD

Blood (2019) 134 (Supplement_1): 1970.

https://doi.org/10.1182/blood-2019-128051

Published: 2019

Journal Articles

An Educational Study Promoting the Delivery of TCD Screening in Paediatric Sickle Cell Disease: A European Multi-Centre Perspective

Baba PD Inusa, MD, Corrina Macmahon, MD, Laura Sainati, MD, Raffaella Colombatti, MD PhD, Maddalena Casale, MD PhD, Patrizia Rampazzo, MD, Silverio Perrotta, Soundrie Padayachee

Blood (2019) 134 (Supplement_1): 2172.

https://doi.org/10.1182/blood-2019-130390

Published: 2019

Journal Articles

Rapid and Robust Mobilization of CD34+ HSCs without G-CSF Following Administration of Mgta-145 Alone or in Combination with Plerixafor

John F. DiPersio, MD PhD, Jonathan Hoggatt, PhD, Steven Devine, MD, Lukasz Biernat, MD, Haley Howell, BS, BA, Veit Schmelmer, PhD, Jason R. Neale, PhD, Anthony E. Boitano, PhD, Michael P. Cooke, PhD, Kevin A. Goncalves, PhD, Glen D. Raffel, MD PhD, Patrick C. Falahee, PhD, Dwight M. Morrow, PhD, John C. Davis, Jr., MD MPH, MS

Blood (2019) 134 (Supplement_1): 1961.

https://doi.org/10.1182/blood-2019-127523

Published: 2019

Journal Articles

Congenital Dyserythropoietic Anemia Type I Due to Biallelic CDAN1 mutations: Report from the Congenital Dyserythropoietic Anemia Registry (CDAR)

Omar Niss, MD, Robert B. Lorsbach, MD PhD, David K. Buchbinder, MD MSHS, Satheesh Chonat, MD, Morgan L. McLemore, MD, Timothy McCavit, MD, Jeffrey H Schwartz, MD, Jessica Meznarich, MD, Katie Seu, Wenying Zhang, MD PhD, Theodosia A. Kalfa, MD PhD

Blood (2019) 134 (Supplement_1): 3521.

https://doi.org/10.1182/blood-2019-128975

Published: 2019

Journal Articles

Identification of Optimal Thalassemia Screening Strategies for Migrant Populations in Thailand: A Mixed-Methods Approach

Julia Z. Xu, MD MSc, Meghan Foe, Wilaslak Tanongsaksakul, MD, Thidarat Suksangpleng, PhD, Supachai Ekwattanakit, PhDMD, Suchada Riolueang, MSc, Marilyn J. Telen, MD, Bonnie N. Kaiser, PhD, Vip Viprakasit, MD DPhil

Blood (2019) 134 (Supplement_1): 2112.

https://doi.org/10.1182/blood-2019-131805

Published: 2019

Journal Articles

Evaluation of Factors Influencing Health Literacy in Adolescents and Adults with Sickle Cell Disease

Nidhi Bhatt, MD, Cecelia Calhoun, MD, Jason R. Hodges, PhD MA, Chinonyelum Nwosu, Guolian Kang, PhD, Allison A. King, MD MPH, PhD, Xiwen Zhao, MSPH, Jane S. Hankins, MD MS

Blood (2019) 134 (Supplement_1): 2110.

https://doi.org/10.1182/blood-2019-130755

Published: 2019

Journal Articles

Characterization of a Genetically Engineered HUDEP2 Cell Line Harboring a Sickle Cell Disease Mutation As a Potential Research Tool for Preclinical Sickle Cell Disease Drug Discovery

Dipti Gupta, Sarah Sturtevant, BSc, Benjamin Vieira, BS, Yukio Nakamura, MD PhD, Sriram Krishnamoorthy, PhD, Melanie Demers, PhD

Blood (2019) 134 (Supplement_1): 3559.

https://doi.org/10.1182/blood-2019-127330

Published: 2019

Journal Articles

Mitapivat (AG-348) in Adults with Pyruvate Kinase Deficiency Who Are Regularly Transfused: A Phase 3, Open-Label, Multicenter, Study (ACTIVATE-T) in Progress

Megan Lynch, MSN, Lei Hua, PhD, Chris Mix, MD, John B. Porter, MA MDFRCP FRCPath

Blood (2019) 134 (Supplement_1): 3526.

https://doi.org/10.1182/blood-2019-123107

Published: 2019

Journal Articles

Disease Model of Atherosclerosis Using Werner Syndrome Patient-Derived Induced Pluripotent Stem Cells Reveals Excessive Inflammatory Response Against Ox-LDL

Sudip Kumar Paul, MSc, Masamitsu Sone, PhD, Hisaya Kato, MD PhD, Yoshiro Maezawa, MD PhD, Yasuo Ouchi, PhD, Koutaro Yokote, MD PhD, Koji Eto, MD, Naoya Takayama, MD PhD

Blood (2019) 134 (Supplement_1): 3615.

https://doi.org/10.1182/blood-2019-123919

Published: 2019

Journal Articles

Preliminary Results of a Prospective Observational Study to Assess the Prevalence of Gaucher Disease in an Adult Population Affected By MGUS

Gaetano Giuffrida, MD, Concetta Conticello, MD, Daniela Nicolosi, PharmD, Valeria Calafiore, Alessandra Romano, MD PhD, Ugo Consoli, MD, Caterina Musolino, MD, Vanessa Innao, Flavia Fiorenza, MD, Cinzia Maugeri, MD, Giuseppe Longo, MD, Maria Rocca Cingari, MD, Francesco Di Raimondo, MD

Blood (2019) 134 (Supplement_1): 4868.

https://doi.org/10.1182/blood-2019-129944

Published: 2019

Journal Articles

How Caring for Toddlers and Young Children with Severe Haemophilia Impacts on Caregiver's Burden

Susan Halimeh, MD, Manuela Siebert, Sylvia Von Mackensen

Blood (2019) 134 (Supplement_1): 3461.

https://doi.org/10.1182/blood-2019-131185

Published: 2019

Journal Articles

Ex Vivo Expansion with E478 Increases the Rate of CRISPR-Mediated Homology Directed Repair of the Beta-Globin Gene in Human Hematopoietic Stem Cells By >10-Fold and Improves In Vivo Engraftment By >120-Fold

Kevin A. Goncalves, PhD, Megan D. Hoban, PhD, Sharon L. Hyzy, MS, Katia S. George, BA, Anthony E. Boitano, PhD, Michael P. Cooke, PhD

Blood (2019) 134 (Supplement_1): 4634.

https://doi.org/10.1182/blood-2019-131157

Published: 2019

Journal Articles

Detection and Analysis of Gene Mutations in Patients with Glanzmann's Thrombasthenia

Boyang Sun, Donglei Zhang, Huiyuan Li, Xueqing Dou, Renchi Yang, MD

Blood (2019) 134 (Supplement_1): 2373.

https://doi.org/10.1182/blood-2019-129446

Published: 2019

Journal Articles

The Mystery of "Magic Blood" - Familial Macrothrombocytopenia Associated with a Novel Variant in GP1BA Gene

Zhaohui Liao Arter, MD, Caitlin Yatogo, MS, CGC, Michael C. Chicka, PhD, Jeffrey L. Berenberg, MD

Blood (2019) 134 (Supplement_1): 2380.

https://doi.org/10.1182/blood-2019-122384

Published: 2019

NARROW

Introduction to a review series on gene therapy and gene editing for sickle cell disease and hemophilia

Pediatric hemophagocytic lymphohistiocytosis

Hemolysis in the Spleen Drives Erythrocyte Turnover

Successful Bone Marrow Transplantation Using an Immunomyelosuppressive Conditioning in Patients with Severe Congenital Neutropenia: The Results of a Single-Institute

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

Social Vulnerability Is Associated with Emergency Department Dependency in Pediatric Sickle Cell Disease Patients

A New Step in Understanding of Fanconi Patients Peripheral Stem Cell Harvesting, a Bridge to Gene Therapy

An Educational Study Promoting the Delivery of TCD Screening in Paediatric Sickle Cell Disease: A European Multi-Centre Perspective

Rapid and Robust Mobilization of CD34+ HSCs without G-CSF Following Administration of Mgta-145 Alone or in Combination with Plerixafor

Congenital Dyserythropoietic Anemia Type I Due to Biallelic CDAN1 mutations: Report from the Congenital Dyserythropoietic Anemia Registry (CDAR)

Identification of Optimal Thalassemia Screening Strategies for Migrant Populations in Thailand: A Mixed-Methods Approach

Evaluation of Factors Influencing Health Literacy in Adolescents and Adults with Sickle Cell Disease

Characterization of a Genetically Engineered HUDEP2 Cell Line Harboring a Sickle Cell Disease Mutation As a Potential Research Tool for Preclinical Sickle Cell Disease Drug Discovery

Mitapivat (AG-348) in Adults with Pyruvate Kinase Deficiency Who Are Regularly Transfused: A Phase 3, Open-Label, Multicenter, Study (ACTIVATE-T) in Progress

Disease Model of Atherosclerosis Using Werner Syndrome Patient-Derived Induced Pluripotent Stem Cells Reveals Excessive Inflammatory Response Against Ox-LDL

Preliminary Results of a Prospective Observational Study to Assess the Prevalence of Gaucher Disease in an Adult Population Affected By MGUS

How Caring for Toddlers and Young Children with Severe Haemophilia Impacts on Caregiver's Burden

Ex Vivo Expansion with E478 Increases the Rate of CRISPR-Mediated Homology Directed Repair of the Beta-Globin Gene in Human Hematopoietic Stem Cells By >10-Fold and Improves In Vivo Engraftment By >120-Fold

Detection and Analysis of Gene Mutations in Patients with Glanzmann's Thrombasthenia

The Mystery of "Magic Blood" - Familial Macrothrombocytopenia Associated with a Novel Variant in GP1BA Gene

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American Society of Hematology

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American Society of Hematology

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