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Journal Articles

Erythroferrone Regulates Bone Remodeling in β-Thalassemia

Melanie Castro-Mollo, MD, Marc Ruiz Martinez, PhD, Maria Feola, PhD, Anisa Azatovna Gumerova, MD, Carla Casu, PhD, Robert E Fleming, MD, Stefano Rivella, PhD, Tony Yuen, PhD, Mone Zaidi, MD PhD, Yelena Ginzburg, MD

Blood (2019) 134 (Supplement_1): 2.

https://doi.org/10.1182/blood-2019-125822

Published: 2019

Journal Articles

PP-14, a Novel Structurally-Enhanced Antisickling Allosteric Hemoglobin Effector, Increases Oxygen Affinity and Disrupts Hemoglobin S Polymer Formation

Martin K. Safo, PhD, Piyusha P Pagare, PhD, Mohini Ghatge, PhD, Carla Casu, PhD, Valentina Ghiaccio, PhD, Nancy Anabaraonye, Xiaomeng Xu, MS, Stefano Rivella, PhD, Patrick C. Hines, MD PhD, Ke Liu, PhD, Yan Zhang, PhD, Jürgen Venitz, MD PhD, Osheiza Abdulmalik, DVM

Blood (2019) 134 (Supplement_1): 73.

https://doi.org/10.1182/blood-2019-128646

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

Absence of TNF-α Promotes Anemia of Inflammation and Inflammatory Bowel Disease

Ritama Gupta, PhD, Lo Presti Vania, Carla Casu, PhD, Rea Oikonomidou, MD, Emir OHara, Stefano Rivella, PhD

Blood (2017) 130 (Supplement 1): 3577.

https://doi.org/10.1182/blood.V130.Suppl_1.3577.3577

Published: 2017

Journal Articles

Developing a Galnac-Conjugated TMPRSS6 Antisense Therapy for the Treatment of β-Thalassemia

Mariam Aghajan, PhD, Carla Casu, PhD, Vania lo Presti, Sheri Booten, Brett P. Monia, PhD, Stefano Rivella, PhD, Shuling Guo, PhD

Blood (2016) 128 (22): 1013.

https://doi.org/10.1182/blood.V128.22.1013.1013

Published: 2016

Journal Articles

Vzhe-039, a Novel Structurally-Enhanced Allosteric Hemoglobin Effector Inhibits Sickling of SS Erythrocytes In Vitro , and Exhibits Improved Pharmacologic Properties In Vivo

Martin K. Safo, PhD, Guoyan Xu, PhD, Mohini Ghatge, PhD, Qiukan Chen, Carla Casu, PhD, David Tanya, PhD, Yan Zhang, PhD, Jurgen Venitz, MD PhD, Osheiza Abdulmalik, DVM, MS

Blood (2016) 128 (22): 3645.

https://doi.org/10.1182/blood.V128.22.3645.3645

Published: 2016

Journal Articles

Administration of Minihepcidins to Animals Affected By β-Thalassemia Major Reduces Anemia and Splenomegaly

Roberta Chessa, PhD, Ritama Gupta, MSc, Carla Casu, PhD, Robert E Fleming, MD, Yelena Ginzburg, Brian MacDonald, MB, ChB, PhD, Stefano Rivella, PhD

Blood (2016) 128 (22): 259.

https://doi.org/10.1182/blood.V128.22.259.259

Published: 2016

Journal Articles

Genetic Investigation of the Role of GDF11 in the Treatment of β-Thalassemia and MDS

Paraskevi Rea Oikonomidou, MD, Ping La, PhD, Ritama Gupta, MSc, Vania Lo Presti, Carla Casu, PhD, Laura Breda, PhD, Deborah Watson, PhD, Rajasekhar NVS Suragani, PhD, Ravindra Kumar, PhD, Stefano Rivella, PhD

Blood (2016) 128 (22): 2439.

https://doi.org/10.1182/blood.V128.22.2439.2439

Published: 2016

Journal Articles

Targeting TMPRSS6 Using Antisense Technology for the Treatment of Beta-Thalassemia

Shuling Guo, PhD, Mariam Aghajan, PhD, Carla Casu, PhD, Sara Gardenghi, PhD, Sheri Booten, Stefano Rivella, PhD, Brett P. Monia, PhD

Blood (2015) 126 (23): 753.

https://doi.org/10.1182/blood.V126.23.753.753

Published: 2015

Journal Articles

Lack of Beta-1 Integrin Limits Stress Erythropoiesis and Splenomegaly in Beta-Thalassemia

Roberta Chessa, PhD, Ritama Gupta, MSc, Bart J Crielaard, MD PhD, Carla Casu, PhD, Rick Feldman, PhD, Stefano Rivella, PhD

Blood (2015) 126 (23): 2196.

https://doi.org/10.1182/blood.V126.23.2196.2196

Published: 2015

Journal Articles

Use of Minihepcidins As a “Medical Phlebotomy” in the Treatment of Polycythemia Vera

Carla Casu, PhD, Rea Oikonomidau, MD, Elizabeta Nemeth, PhD, Tomas Ganz, MD PhD, Brian MacDonald, Stefano Rivella, PhD

Blood (2014) 124 (21): 3231.

https://doi.org/10.1182/blood.V124.21.3231.3231

Published: 2014

Journal Articles

Beta-1 Integrin Controls Homing and Expansion of Erythroid Cells in Stress Erythropoiesis and ß-Thalassemia

Bart Crielaard, Roberta Chessa, PhD, Ritama Gupta, Carla Casu, PhD, Stefano Rivella, PhD

Blood (2014) 124 (21): 2661.

https://doi.org/10.1182/blood.V124.21.2661.2661

Published: 2014

Journal Articles

Concurrent Treatment with Minhepcidin and Deferiprone Improves Anemia and Enhances Reduction of Spleen Iron in a Mouse Model of Non-Transfusion Dependent Thalassemia

Carla Casu, PhD, Rea Oikonomidau, MD, Yatrik Shah, PhD, Elizabeta Nemeth, PhD, Tomas Ganz, MD PhD, Brian MacDonald, Stefano Rivella, PhD

Blood (2014) 124 (21): 748.

https://doi.org/10.1182/blood.V124.21.748.748

Published: 2014

Journal Articles

Combination of Tmprss6 -ASO and the Iron Chelator Deferiprone Improves Erythropoiesis and Reduces Iron Overload in a Mouse Model of Beta-Thalassemia

Carla Casu, PhD, Mariam Aghajan, PhD, Rea Oikonomidau, MD, Shuling Guo, PhD, Brett P. Monia, PhD, Stefano Rivella, PhD

Blood (2014) 124 (21): 4024.

https://doi.org/10.1182/blood.V124.21.4024.4024

Published: 2014

Journal Articles

Macrophages Regulate Stress Erythropoiesis Through Direct Cellular Interactions Associated With Integrin β1-Focal Adhesion Kinase Signaling

Bart J Crielaard, MD, PhD, Carla Casu, PhD, Pedro Ramos, PhD, Sara Gardenghi, PhD, Laura Breda, PhD, Ritama Gupta, Stefano Rivella, PhD

Blood (2013) 122 (21): 307.

https://doi.org/10.1182/blood.V122.21.307.307

Published: 2013

Journal Articles

Distinct Roles For Hepcidin and Interleukin 6 In The Recovery From Anemia Following Administration Of Heat-Killed Brucella Abortus

Sara Gardenghi, PhD, Thomas M. Renaud, MD, Alessandra Meloni, PhD, Carla Casu, PhD, Bart J. Crielaard, PhD, Laura M. Bystrom, PhD, Noa Greenberg, MD, Barbra J. Sasu, PhD, Keegan S. Cooke, Stefano Rivella, PhD

Blood (2013) 122 (21): 430.

https://doi.org/10.1182/blood.V122.21.430.430

Published: 2013

Journal Articles

Treatment With Minihepcidin Peptide Improves Anemia and Iron Overload In a Mouse Model Of Thalassemia Intermedia

Carla Casu, PHD, Adam Goldberg, Elizabeta Nemeth, PhD, Tomas Ganz, MD, PhD, Sara Gardenghi, PhD, Brian MacDonald, Stefano Rivella, PhD

Blood (2013) 122 (21): 431.

https://doi.org/10.1182/blood.V122.21.431.431

Published: 2013

Journal Articles

Removal of Macrophages From the Erythroid Niche Impairs Stress Erythropoiesis but Improves Pathophysiology of Polycythemia Vera and Beta-Thalassemia

Pedro Ramos, PhD, Carla Casu, PhD, Sara Gardenghi, PhD, Laura Breda, Ph.D, Bart J Crielaard, PhD, Ritama Gupta, Undergraduate student, Ella Guy, Maria F Mariongiu, PhD, Ross L. Levine, Omar Abdel-Wahab, PhD, Benjamin L. Ebert, Nico van Rooijen, PhD, Saghi Ghaffari, MD, PhD, Robert W Grady, PhD, Patricia Giardina, MD, Stefano Rivella, PhD

Blood (2012) 120 (21): 81.

https://doi.org/10.1182/blood.V120.21.81.81

Published: 2012

Journal Articles

Potential Therapeutic Applications of Jak2 Inhibitors in Beta-Thalassemia and Sickle Cell Disease,

Carla Casu, PhD, Pedro Ramos, PhD, Luca Melchiori, PhD, Ella Guy, Eliezer A. Rachmilewitz, Patricia Giardina, MD, Robert W Grady, PhD, Maria de Sousa, MD, PhD, Stefano Rivella, PhD

Blood (2011) 118 (21): 3187.

https://doi.org/10.1182/blood.V118.21.3187.3187

Published: 2011

NARROW

Erythroferrone Regulates Bone Remodeling in β-Thalassemia

PP-14, a Novel Structurally-Enhanced Antisickling Allosteric Hemoglobin Effector, Increases Oxygen Affinity and Disrupts Hemoglobin S Polymer Formation

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

Absence of TNF-α Promotes Anemia of Inflammation and Inflammatory Bowel Disease

Developing a Galnac-Conjugated TMPRSS6 Antisense Therapy for the Treatment of β-Thalassemia

Vzhe-039, a Novel Structurally-Enhanced Allosteric Hemoglobin Effector Inhibits Sickling of SS Erythrocytes In Vitro , and Exhibits Improved Pharmacologic Properties In Vivo

Administration of Minihepcidins to Animals Affected By β-Thalassemia Major Reduces Anemia and Splenomegaly

Genetic Investigation of the Role of GDF11 in the Treatment of β-Thalassemia and MDS

Targeting TMPRSS6 Using Antisense Technology for the Treatment of Beta-Thalassemia

Lack of Beta-1 Integrin Limits Stress Erythropoiesis and Splenomegaly in Beta-Thalassemia

Use of Minihepcidins As a “Medical Phlebotomy” in the Treatment of Polycythemia Vera

Beta-1 Integrin Controls Homing and Expansion of Erythroid Cells in Stress Erythropoiesis and ß-Thalassemia

Concurrent Treatment with Minhepcidin and Deferiprone Improves Anemia and Enhances Reduction of Spleen Iron in a Mouse Model of Non-Transfusion Dependent Thalassemia

Combination of Tmprss6 -ASO and the Iron Chelator Deferiprone Improves Erythropoiesis and Reduces Iron Overload in a Mouse Model of Beta-Thalassemia

Macrophages Regulate Stress Erythropoiesis Through Direct Cellular Interactions Associated With Integrin β1-Focal Adhesion Kinase Signaling

Distinct Roles For Hepcidin and Interleukin 6 In The Recovery From Anemia Following Administration Of Heat-Killed Brucella Abortus

Treatment With Minihepcidin Peptide Improves Anemia and Iron Overload In a Mouse Model Of Thalassemia Intermedia

Removal of Macrophages From the Erythroid Niche Impairs Stress Erythropoiesis but Improves Pathophysiology of Polycythemia Vera and Beta-Thalassemia

Potential Therapeutic Applications of Jak2 Inhibitors in Beta-Thalassemia and Sickle Cell Disease,

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American Society of Hematology

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American Society of Hematology

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