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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

Mitapivat (AG-348) in Adults with Pyruvate Kinase Deficiency Who Are Not Regularly Transfused: A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study (ACTIVATE) in Progress

Eduard J. van Beers, MD PhD, Malia P. Judge, BS, RN, Lei Hua, PhD, Chris Mix, MD

Blood (2019) 134 (Supplement_1): 4791.

https://doi.org/10.1182/blood-2019-123100

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

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

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

High Levels of Peripheral Vasoconstriction Detected By Polysomnography Predict More Acute Severe Pain Episodes in Children with Sickle Cell Anemia

Patjanaporn Chalacheva, PhD, Yunhua Ji, MS, Carol L. Rosen, MD, Michael R. DeBaun, MDMPH, Michael C.K. Khoo, PhD, Thomas Coates, MD

Blood (2019) 134 (Supplement_1): 894.

https://doi.org/10.1182/blood-2019-121944

Published: 2019

Journal Articles

Northstar-3: Interim Results from a Phase 3 Study Evaluating Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Either a β0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene

Ashutosh Lal, MD, Franco Locatelli, MD PhD, Janet L. Kwiatkowski, MD MSCE, Andreas E. Kulozik, MD PhD, Evangelia Yannaki, MD, John B. Porter, MA MDFRCP FRCPath, Isabelle Thuret, MD, Martin G. Sauer, MD, Heidi Elliot, Ying Chen, PhD, Richard A. Colvin, MD PhD, Alexis A. Thompson, MD MPH

Blood (2019) 134 (Supplement_1): 815.

https://doi.org/10.1182/blood-2019-128482

Published: 2019

Journal Articles

Home-Based Assessment of Patient Reported Outcome Measures Using a Smartphone App Platform: A Feasibility Study

Sherif M. Badawy, MD, Leonardo Barrera, MPH, Graham Zolkowski, BS, Zeinab Alward, MBChB, MPH

Blood (2019) 134 (Supplement_1): 420.

https://doi.org/10.1182/blood-2019-131334

Published: 2019

Journal Articles

Decline in Forced Expiratory Volume in 1 Second in Older Adults with Sickle Cell Disease Is Similar to Adults with Cystic Fibrosis

Brock Hodges, Jodi-Anne T Stewart, BS, Mark Rodeghier, PhD, Karina L Wilkerson, NP, MSN, Adetola A. Kassim, MDMS, Michael R. DeBaun, MDMPH

Blood (2019) 134 (Supplement_1): 1012.

https://doi.org/10.1182/blood-2019-123428

Published: 2019

Journal Articles

Management Strategies and Satisfaction Levels in Patients with Sickle Cell Disease: Interim Results from the International Sickle Cell World Assessment Survey (SWAY)

John James, MSc, Biree Andemariam, MD, Baba PD Inusa, MD, Fuad El-Rassi, Beverly Francis-Gibson, Alecia C. Nero, MD, Caterina P. Minniti, MD, Cassandra Trimnell, BA, Miguel R. Abboud, MD, Jean-Benoît Arlet, Raffaella Colombatti, Mariane de Montalembert, MD PhD, Suman Jain, Wasil Jastaniah, Erfan Nur, MD PhD, Laurie DeBonnett, MD, Ifeyinwa (ify) Osunkwo, MD MPH

Blood (2019) 134 (Supplement_1): 1017.

https://doi.org/10.1182/blood-2019-123643

Published: 2019

Journal Articles

The Effect of Crizanlizumab Plus Standard of Care (SoC) Versus Soc Alone on Renal Function in Patients with Sickle Cell Disease and Chronic Kidney Disease: A Randomized, Multicenter, Open-Label, Phase II Study (STEADFAST)

Kenneth I. Ataga, MD, Santosh L. Saraf, MD, Vimal K. Derebail, MD, Claire C. Sharpe, Adlette Inati, MD, Jeffrey D. Lebensburger, DO, MSPH, Laurie DeBonnett, MD, Shirong Zhang, Pablo Bartolucci, MD PhD

Blood (2019) 134 (Supplement_1): 1018.

https://doi.org/10.1182/blood-2019-124823

Published: 2019

Journal Articles

Blinatumomab in Pediatric Patients with Relapsed/Refractory B-Cell Precursor and Molecularly Resistant Acute Lymphoblastic Leukemia (R/R ALL): Updated Analysis of 110 Patients Treated in an Expanded Access Study (RIALTO)

Franco Locatelli, MD, Gerhard Zugmaier, Peter Bader, MD, Sima Jeha, MD, Paul-Gerhardt Schlegel, Jean-Pierre Bourquin, MD PhD, Rupert Handgretinger, MD, Benoit Brethon, MD, Claudia Rossig, Christiane Chen-Santel

Blood (2019) 134 (Supplement_1): 1294.

https://doi.org/10.1182/blood-2019-121615

Published: 2019

Journal Articles

Pediatric Patient with Hypodysfibrinogenemia Presenting with an Unprovoked Portal Vein Thrombosis

Brenton J Francisco, MD, Balkrishan Sharma, PhD, Phillip A Owens, PhD, Hannah Russell, Joseph S. Palumbo, MD

Blood (2019) 134 (Supplement_1): 1145.

https://doi.org/10.1182/blood-2019-123760

Published: 2019

Journal Articles

Correlation of Voxelotor Exposure with Hemoglobin Response and Measures of Hemolysis in Patients from the HOPE Study

Jo Howard, MB, BChir, Elliott Vichinsky, MD UCSF, Jennifer Knight-Madden, MBBS, PhD, Margaret Tonda, PharmD, Carla Washington, Barbara Tong, PhD, Joshua Lehrer-Graiwer, MD MPhil, FACC, Victor R Gordeuk, MD

Blood (2019) 134 (Supplement_1): 1020.

https://doi.org/10.1182/blood-2019-130802

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

CD244 Marks Non-Functional Hematopoietic Stem Cells with a Mast Cell Signature after Induction of Endoplasmic Reticulum Stress

Valgardur Sigurdsson, PhD, Shuhei Koide, PhD, Visnja Radulovic, PhD, Els Mansell, PhD, Mark Van Der Garde, MSc, PhD, Tiit Örd, PhD, Tonis Örd, PhD, Kenichi Miharada, PhD

Blood (2019) 134 (Supplement_1): 2474.

https://doi.org/10.1182/blood-2019-128239

Published: 2019

Journal Articles

Predictors of Disease Progression and Survival in Patients with Myelodysplastic Syndrome Secondary to Inherited Bone Marrow Failure Syndromes

Blood (2019) 134 (Supplement_1): 2507.

https://doi.org/10.1182/blood-2019-130129

Published: 2019

Journal Articles

Modeling of Immune Reconstitution Post CD34 Selected Stem Cell Transplantation in Pediatric Patients with Severe Combined Immune Deficiency

Jean-Sebastien Diana, MD, Naim Bouazza, PhD, Chloé Couzin, MD, Martin Castelle, MD, Alessandra Magnani, MD PhD, Elisa Magrin, PhD, Laure Joseph, MD, Marianne Delville, MD, Francois Lefrere, MD, jean-Marc Treluyer, MD PhD, Capucine Picard, MD PhD, Despina Moshous, MD PhD, Isabelle André, PhD, Stephane Blanche, MD, Benedicte Neven, MD PhD, Marina Cavazzana, MD PhD

Blood (2019) 134 (Supplement_1): 5668.

https://doi.org/10.1182/blood-2019-127974

Published: 2019

Journal Articles

CRISPR/dCas9 (D10A) -Mediated Hb CS Gene Editing and Identification of Genetically Modified Fibroblasts

Lian Yu, Wei Hao Wu, WANG Shunqing, Ken H. Young, Funeng Jiang, PhD

Blood (2019) 134 (Supplement_1): 5754.

https://doi.org/10.1182/blood-2019-129931

Published: 2019

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

Pediatric hemophagocytic lymphohistiocytosis

Mitapivat (AG-348) in Adults with Pyruvate Kinase Deficiency Who Are Not Regularly Transfused: A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study (ACTIVATE) in Progress

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

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

Hemolysis in the Spleen Drives Erythrocyte Turnover

High Levels of Peripheral Vasoconstriction Detected By Polysomnography Predict More Acute Severe Pain Episodes in Children with Sickle Cell Anemia

Northstar-3: Interim Results from a Phase 3 Study Evaluating Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Either a β0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene

Home-Based Assessment of Patient Reported Outcome Measures Using a Smartphone App Platform: A Feasibility Study

Decline in Forced Expiratory Volume in 1 Second in Older Adults with Sickle Cell Disease Is Similar to Adults with Cystic Fibrosis

Management Strategies and Satisfaction Levels in Patients with Sickle Cell Disease: Interim Results from the International Sickle Cell World Assessment Survey (SWAY)

The Effect of Crizanlizumab Plus Standard of Care (SoC) Versus Soc Alone on Renal Function in Patients with Sickle Cell Disease and Chronic Kidney Disease: A Randomized, Multicenter, Open-Label, Phase II Study (STEADFAST)

Blinatumomab in Pediatric Patients with Relapsed/Refractory B-Cell Precursor and Molecularly Resistant Acute Lymphoblastic Leukemia (R/R ALL): Updated Analysis of 110 Patients Treated in an Expanded Access Study (RIALTO)

Pediatric Patient with Hypodysfibrinogenemia Presenting with an Unprovoked Portal Vein Thrombosis

Correlation of Voxelotor Exposure with Hemoglobin Response and Measures of Hemolysis in Patients from the HOPE Study

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

CD244 Marks Non-Functional Hematopoietic Stem Cells with a Mast Cell Signature after Induction of Endoplasmic Reticulum Stress

Predictors of Disease Progression and Survival in Patients with Myelodysplastic Syndrome Secondary to Inherited Bone Marrow Failure Syndromes

Modeling of Immune Reconstitution Post CD34 Selected Stem Cell Transplantation in Pediatric Patients with Severe Combined Immune Deficiency

CRISPR/dCas9 (D10A) -Mediated Hb CS Gene Editing and Identification of Genetically Modified Fibroblasts

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