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Journal Articles

Hemoglobin S Polymerization Can be Inhibited By Alterations in Erythrocyte Glycolysis

William R. Simmons, Quan Li, PhD, John A. Hausman, Suilan Zheng, PhD, William Eaton, MD, Philip S. Low, PhD, David M. Bodine, PhD

Blood (2019) 134 (Supplement_1): 977.

https://doi.org/10.1182/blood-2019-121709

Published: 2019

Journal Articles

Sickle Cell Hemoglobin Impairs Lysosomal Processing in Macrophages Leading to Inflammasome Activation and Inflammation

Marina Jerebtsova, PhD, Xiaomei Niu, MD, Sharmin Diaz, NP, Namita Kumari, PhD, Sergei Nekhai, PhD

Blood (2019) 134 (Supplement_1): 979.

https://doi.org/10.1182/blood-2019-130741

Published: 2019

Journal Articles

Understanding Heterogeneity of Fetal Hemoglobin Induction through Comparative Analysis of Stage-Matched F- and a-Cells

Eugene Khandros, MD PhD, Peng Huang, PhD, Scott A. Peslak, MD PhD, Belinda Giardine, Zhe Zhang, PhD, Cheryl A Keller, PhD, Ross C. Hardison, PhD, Gerd A. Blobel, MD PhD

Blood (2019) 134 (Supplement_1): 981.

https://doi.org/10.1182/blood-2019-124099

Published: 2019

Journal Articles

Exploring the Drivers of Potential Clinical Benefit in Initial Patients Treated in the Hgb-206 Study of Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy

Mark C. Walters, MD, John F. Tisdale, MD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Markus Y. Mapara, MD PhD, Manfred Schmidt, PhD, Alexandra L. Miller, Francis J. Pierciey, Jr., Wenmei Huang, PhD MS, Jean-Antoine Ribeil, MD PhD, Julie Kanter, MD, Alexis A. Thompson, MD MPH

Blood (2019) 134 (Supplement_1): 2061.

https://doi.org/10.1182/blood-2019-128814

Published: 2019

Journal Articles

Deletion of TLR4 in Townes SS Mice Prevents Microvascular Stasis in Response to Hemin, LPS and Hypoxia/Reoxygenation: Role of Inflammation

John D Belcher, PhD, Fuad Abdulla, Chunsheng Chen, Ping Zhang, PhD, Dormarie Rivera-Rodriguez, Rachel Kirchner, Julia Nguyen, Zachary Monroe Kiser, BS, PhD, Kristine Nachbor, Jacob Lloyd, MD, Gregory M Vercellotti, MD

Blood (2019) 134 (Supplement_1): 3555.

https://doi.org/10.1182/blood-2019-127114

Published: 2019

Journal Articles

Rituximab for Preventing Delayed Hemolytic Transfusion Reaction (DHTR) in Sickle CELL Adult Patients: Outcome of Transfusion and SIDE Effects in 58 CASES

Fabian Zanchetta-Balint, France Pirenne, MD PhD, Marc Michel, Armand Mekontso-Dessap, MD PhD, Matthieu Mahevas, MD, Constance Guillaud, MD, Keyvan Razazi, MD, Elena Foïs, MD, Frédéric Galactéros, MD PhD, Pablo Bartolucci, MD PhD, Anoosha Habibi

Blood (2019) 134 (Supplement_1): 3687.

https://doi.org/10.1182/blood-2019-130874

Published: 2019

Journal Articles

Northstar-2: Updated Safety and Efficacy Analysis of Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Non-β0/β0 Genotypes

Alexis A. Thompson, MD MPH, Mark C. Walters, MD, Janet L. Kwiatkowski, MD MSCE, Suradej Hongeng, MD, John B. Porter, MA MDFRCP FRCPath, Martin G. Sauer, MD, Adrian J. Thrasher, MBBS, PhD FMedSci, Isabelle Thuret, MD, Heidi Elliot, Ge Tao, PhD, Richard A. Colvin, MD PhD, Franco Locatelli, MD PhD

Blood (2019) 134 (Supplement_1): 3543.

https://doi.org/10.1182/blood-2019-126046

Published: 2019

Journal Articles

ZNF802 (JAZF1), a Possible New Therapeutic Target for Treatment of β-Thalassemia

Chokdee Wongborisuth, MSc, Amornrat Tangprasittipap, PhD, Usanarat Anurathapan, MD, Nuankanya Sathirapongsasuti, MD, PhD, Orapan Sripichai, PhD, Duantida Songdej, Suradej Hongeng, MD

Blood (2019) 134 (Supplement_1): 3546.

https://doi.org/10.1182/blood-2019-127678

Published: 2019

Journal Articles

Chronic Kidney Disease Is Under-Screened in SCD and Mild Albuminuria Is Associated with a Drop in Hemoglobin: A Report from the Grndad Sickle Cell Registry

Elizabeth Williams, BA, Elizabeth Brown, Deepa Manwani, MD, Payal Desai, MD, Joshua J. Field, MD, Lynne D. Neumayr, Susan Padrino, MD, E. Leila Jerome Clay, MD MCTS, FAAP, Ze Cong, PhD, Irene Agodoa, MD, Carolyn Hoppe, MD, Sophie M. Lanzkron, MD, Jane A. Little, MD

Blood (2019) 134 (Supplement_1): 2284.

https://doi.org/10.1182/blood-2019-124933

Published: 2019

Journal Articles

The Relationships between Target Gene Transduction, Engraftment of HSCs and RBC Physiology in Sickle Cell Disease Gene Therapy

Melissa Bonner, PhD, Julie Kanter, MD, Elizabeth Macari, PhD, Ricky Lane, Gretchen Lewis, BMBS, Paige Coles, Sarah Kassenaar, Sai Mynampati, MS, Robert Schulze, Madison Hebert, Mark C. Walters, MD, Alexis A. Thompson, MD MPH, Mohammed Asmal, MD PhD, John F. Tisdale, MD, Francis J. Pierciey, Jr.

Blood (2019) 134 (Supplement_1): 206.

https://doi.org/10.1182/blood-2019-129124

Published: 2019

Journal Articles

Cannabidiol Reduces Acute and Chronic Hyperalgesia in Humanized Sickle Mice

Varun Sagi, BS, Jianxun Lei, PhD, Stacy B Kiven, BS, Huy Tran, BS, Kalpna Gupta, PhD

Blood (2019) 134 (Supplement_1): 77.

https://doi.org/10.1182/blood-2019-129706

Published: 2019

Journal Articles

Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies

Laura Breda, PhD, Valentina Ghiaccio, PhD, Silvia Pires Lourenco, MSc, Danuta Jadwiga Jarocha, PhD, Yasuhiro Ikawa, MD PhD, Naoto Tanaka, PhD, Virginia Guzikowski, Carla Casu, PhD, Kim Smith-Whitley, MD, Janet L. Kwiatkowski, MD MSCE, Stefano Rivella, PhD

Blood (2019) 134 (Supplement_1): 2242.

https://doi.org/10.1182/blood-2019-124043

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

Blood (2019) 134 (Supplement_1): 2244.

https://doi.org/10.1182/blood-2019-127017

Published: 2019

Journal Articles

Treatment of Delayed Hemolytic Transfusion Reactions in Sickle Cell Disease Patients By an Anti-C5 Antibody

Blood (2019) 134 (Supplement_1): 2458.

https://doi.org/10.1182/blood-2019-127176

Published: 2019

Journal Articles

Results from the Completed Hgb-205 Trial of Lentiglobin for β-Thalassemia and Lentiglobin for Sickle Cell Disease Gene Therapy

Blood (2019) 134 (Supplement_1): 3358.

https://doi.org/10.1182/blood-2019-127393

Published: 2019

Journal Articles

High-Throughput RNA Sequencing Analysis Reveals Distinct Molecular Signature in NRAS and PTPN11 JMML Patients

Pier Paolo Leoncini, PhD, Alessandro Domenico Silvestris, PhD, Patrizia Vitullo, PhD, Marco Zecca, MD, Christian Flotho, MD PhD, Charlotte M. Niemeyer, MD, Angela Gallo, PhD, Alice Bertaina, MD PhD, Franco Locatelli, MD PhD, Pietro Merli, MD

Blood (2019) 134 (Supplement_1): 4229.

https://doi.org/10.1182/blood-2019-129965

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

Hydroxyurea Reverses Dysfunctional Ubiquitin-Proteasomal System in Sickle CELL Disease and Suppresses Posttranslational Alterations in Hemoglobin and CELL Membranes

Swee Lay Thein, DSc, MBBS, Arun S Shet, MD, Michael Brad Strader, PhD, Fanato Meng, PhD, Michael Heaven, PhD, Jana Sirsendu, PhD, Abdu Alayash, PhD

Blood (2019) 134 (Supplement_1): 4822.

https://doi.org/10.1182/blood-2019-122840

Published: 2019

Journal Articles

Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study

Janet L. Kwiatkowski, MD MSCE, Alexis A. Thompson, MD MPH, John E.J. Rasko, MBBS, PhD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, MD, Marina Cavazzana, MD PhD, P. Joy Ho, MBBS DPhil FRACP FRCPA, Manfred Schmidt, PhD, Morris Kletzel, MD MBA, Elliott P. Vichinsky, MD, Briana Deary, Ying Chen, PhD, Alexandria Petrusich, Mark C. Walters, MD

Blood (2019) 134 (Supplement_1): 4628.

https://doi.org/10.1182/blood-2019-125807

Published: 2019

Journal Articles

Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study

Julie Kanter, MD, John F. Tisdale, MD, Markus Y. Mapara, MD PhD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Manfred Schmidt, PhD, Alexandra L. Miller, Francis J. Pierciey, Jr., Wenmei Huang, PhD MS, Jean-Antoine Ribeil, MD PhD, Alexis A. Thompson, MD MPH, Mark C. Walters, MD

Blood (2019) 134 (Supplement_1): 990.

https://doi.org/10.1182/blood-2019-128894

Published: 2019

NARROW

Hemoglobin S Polymerization Can be Inhibited By Alterations in Erythrocyte Glycolysis

Sickle Cell Hemoglobin Impairs Lysosomal Processing in Macrophages Leading to Inflammasome Activation and Inflammation

Understanding Heterogeneity of Fetal Hemoglobin Induction through Comparative Analysis of Stage-Matched F- and a-Cells

Exploring the Drivers of Potential Clinical Benefit in Initial Patients Treated in the Hgb-206 Study of Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy

Deletion of TLR4 in Townes SS Mice Prevents Microvascular Stasis in Response to Hemin, LPS and Hypoxia/Reoxygenation: Role of Inflammation

Rituximab for Preventing Delayed Hemolytic Transfusion Reaction (DHTR) in Sickle CELL Adult Patients: Outcome of Transfusion and SIDE Effects in 58 CASES

Northstar-2: Updated Safety and Efficacy Analysis of Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Non-β0/β0 Genotypes

ZNF802 (JAZF1), a Possible New Therapeutic Target for Treatment of β-Thalassemia

Chronic Kidney Disease Is Under-Screened in SCD and Mild Albuminuria Is Associated with a Drop in Hemoglobin: A Report from the Grndad Sickle Cell Registry

The Relationships between Target Gene Transduction, Engraftment of HSCs and RBC Physiology in Sickle Cell Disease Gene Therapy

Cannabidiol Reduces Acute and Chronic Hyperalgesia in Humanized Sickle Mice

Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies

2'-O-Methoxyethyl Splice-Switching Oligos to Reverse Splicing from IVS2-745 β-Thalassemia Patient Cells: A Foundation for Potential Therapies

Treatment of Delayed Hemolytic Transfusion Reactions in Sickle Cell Disease Patients By an Anti-C5 Antibody

Results from the Completed Hgb-205 Trial of Lentiglobin for β-Thalassemia and Lentiglobin for Sickle Cell Disease Gene Therapy

High-Throughput RNA Sequencing Analysis Reveals Distinct Molecular Signature in NRAS and PTPN11 JMML Patients

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

Hydroxyurea Reverses Dysfunctional Ubiquitin-Proteasomal System in Sickle CELL Disease and Suppresses Posttranslational Alterations in Hemoglobin and CELL Membranes

Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study

Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study

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American Society of Hematology

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American Society of Hematology

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