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Journal Articles

TMPRSS6 Antisense Oligonucleotide Therapy Reverses Inflammation in Jak2 V617F Mutant Mice

Maayan Levy, Huihui Li, Andrew Dunbar, Zixing Huang, Young Park, Min Lu, Pinanong Na Phattalung, Marina Planoutene, Carla Casu, Stefano Rivella, Shuling Guo, Ross L Levine, Ronald Hoffman, Yelena Ginzburg

Blood (2024) 144 (Supplement 1): 8.

https://doi.org/10.1182/blood-2024-203806

Published: 2024

Journal Articles

Combination of a TGF-β Ligand Trap (RAP-GRL) and TMPRSS6 -ASO Is Superior for Correcting β-Thalassemia

Amaliris Guerra, Perry Demsko, Ariel Rivera, Carlo Castruccio Castracani, Laura Breda, Naoto Tanaka, Shuling Guo, Stefano Rivella

Blood (2022) 140 (Supplement 1): 1944–1945.

https://doi.org/10.1182/blood-2022-166801

Published: 2022

Journal Articles

Dissecting the Mechanisms of Hepcidin and BMP-SMAD Pathway Regulation By FKBP12

Alessia Pagani, Mariateresa Pettinato, Alessandro Dulja, Silvia Colucci, Mariam Aghajan, Valeria Furiosi, Martina U. Muckenthaler, Shuling Guo, Antonella Nai, Laura Silvestri

Blood (2021) 138 (Supplement 1): 2008.

https://doi.org/10.1182/blood-2021-152172

Published: 2021

Journal Articles

Combination of a Luspatercept-like Drug (RAP-GRL) and Tmprss6 -ASO Is Superior to Either Drug Alone for Correcting β-Thalassemia

Amaliris Guerra, Perry Demsko, Paige McVeigh, Sayantani Sinha, Carlo Castruccio Castracani, Laura Breda, Carla Casu, Shuling Guo, Stefano Rivella

Blood (2021) 138 (Supplement 1): 2013.

https://doi.org/10.1182/blood-2021-151181

Published: 2021

Journal Articles

Rebalancing Iron Homeostasis and Erythropoiesis through Tfr2 Inhibition for Correction of Anemia in CKD

Violante Olivari, Maria Rosa Lidonnici, Mariam Aghajan, Mariateresa Pettinato, Simona Maria Di Modica, Alessia Pagani, Francesca Tiboni, Shuling Guo, Laura Silvestri, Giuliana Ferrari, Antonella Nai

Blood (2021) 138 (Supplement 1): 940.

https://doi.org/10.1182/blood-2021-148384

Published: 2021

Journal Articles

Correcting β-thalassemia by combined therapies that restrict iron and modulate erythropoietin activity

Blood (2020) 136 (17): 1968–1979.

https://doi.org/10.1182/blood.2019004719

Published: 2020

Includes: Supplemental data

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

Targeting the BMP Receptor Inhibitor FKBP12 to Upregulate Hepcidin in Wild-Type and Hemojuvelin-Hemochromatosis Models

Mariateresa Pettinato, Mariam Aghajan, PhD, Violante Olivari, MSc, Alessandro Dulja, MSc, Antonella Nai, PhD, Jessica Bordini, PhD, Alessandro Campanella, PhD, Alessia Pagani, PhD, Shuling Guo, PhD, Clara Camaschella, MD, Laura Silvestri, PhD

Blood (2019) 134 (Supplement_1): 955.

https://doi.org/10.1182/blood-2019-126496

Published: 2019

Journal Articles

Correcting Non-Transfusion Dependent β-Thalassemia by Utilizing a Combined Therapy that Modulates EPO Activity by Limiting Erythroid Cellular Iron Intake

Carla Casu, Vania Lo Presti, Mariam Aghajan, Alison Liu, Kevin A Munoz, Emir O'Hara, Garry Neil, Reem Miari, Hagit Domev, Nir Shapir, Shuling Guo, Stefano Rivella

Blood (2018) 132 (Supplement 1): 164.

https://doi.org/10.1182/blood-2018-164

Published: 2018

Journal Articles

Transmembrane Protease, Serine 6 (TMPRSS6) Antisense Oligonucleotide (IONIS-TMPRSS6-L RX ) Reduces Plasma Iron Levels of Healthy Volunteers in a Phase 1 Clinical Study

Michael McCaleb, PhD, Jason Lickliter, MBBS, PhD, Andrew Dibble, PhD, Eugene Schneider, MD, Mariam Aghajan, Shuling Guo, Steve Hughes, MD MBA, Richard S Geary, PhD, Brett P. Monia, PhD

Blood (2018) 132 (Supplement 1): 3634.

https://doi.org/10.1182/blood-2018-99-115339

Published: 2018

Journal Articles

Inhibiting the Immunophilin FKBP12: A Potential Therapeutic Approach to Iron Overload/Low Hepcidin Disorders

Mariateresa Pettinato, MSc, Mariam Aghajan, Alessandro Dulja, Antonella Nai, PhD, Violante Olivari, Jessica Bordini, MSc, Alessandro Campanella, PhD, Alessia Pagani, PhD, Shuling Guo, Clara Camaschella, MD, Laura Silvestri, PhD

Blood (2018) 132 (Supplement 1): 2333.

https://doi.org/10.1182/blood-2018-99-118112

Published: 2018

Journal Articles

Potential Therapeutic Applications of Jak2 Inhibitors and Hif2a-ASO for the Treatment of β-Thalassemia Intermedia and Major

Carla Casu, Paraskevi Rea Oikonomidou, MD, Vania Lo Presti, Mariam Aghajan, PhD, Shuling Guo, PhD, Abdulmalik Osheiza, Luca Melchiori, Pedro Ramos, Stefano Rivella, PhD

Blood (2016) 128 (22): 1012.

https://doi.org/10.1182/blood.V128.22.1012.1012

Published: 2016

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

Adult Hemoglobin Production, Chain Rebalance, and Splice Correction in IVS2-745 Beta-Thalassemia Patient Cells Using 2'-O-Methoxyethyl Splice-Switching Oligos

Alisa Cheung Dong, Valentina Ghiaccio, Irene Motta, MD, Shuling Guo, PhD, Raechel Peralta, MS, Coralea Stephanou, PhD, Paola Delbini, PhD, Connie Chen, Soteroula Christou, Marina Kleanthous, PhD, Maria Domenica Cappellini, MD, Osheiza Abdulmalik, DVM, MS, Laura Breda, PhD, Stefano Rivella, PhD

Blood (2016) 128 (22): 1014.

https://doi.org/10.1182/blood.V128.22.1014.1014

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

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

Novel Targeted Therapy for Precursor B Cell Acute Lymphoblastic Leukemia: Anti-CD22 Antibody-MXD3 Antisense Oligonucleotide Conjugates

Noriko Satake, MD, Connie Duong, BS, Michael Oestergaard, Sakiko Yoshida, MDPhD, Cathy Chen, Jan A. Nolta, PhD, Shuling Guo, PhD, Punit Seth, PhD, Nitin Nitin, PhD, Joseph Tuscano, MD

Blood (2014) 124 (21): 3719.

https://doi.org/10.1182/blood.V124.21.3719.3719

Published: 2014

Journal Articles

Targeting KLF1 for the Treatment of Sickle Cell Disease Using Antisense Oligonucleotides

Raechel Peralta, MS, Audrey Low, Sheri Booten, Dewang Zhou, Aneeza Kim, Sue Freier, Shuling Guo, PhD, Sue Murray, Tim M. Townes, PhD, Gene Hung

Blood (2014) 124 (21): 4038.

https://doi.org/10.1182/blood.V124.21.4038.4038

Published: 2014

Journal Articles

Targeting BCL11A and KLF1 For The Treatment Of Sickle Cell Disease and β-Thalassemia In Vitro using Antisense Oligonucleotides

Raechel Peralta, Audrey Low, Aneeza Kim, Sue Murray, Shuling Guo, PhD, Sue Freier, Tim M. Townes, PhD, Gene Hung

Blood (2013) 122 (21): 1022.

https://doi.org/10.1182/blood.V122.21.1022.1022

Published: 2013

Journal Articles

Target TMPRSS6 Using Antisense Technology for the Treatment of Hereditary Hemochromatosis and β-Thalassemia

Shuling Guo, PhD, Carla Casu, Sara Gardenghi, PhD, Sheri Booten, Andy Watt, Susan Freier, Ph.D, Stefano Rivella, PhD, Brett P. Monia, PhD

Blood (2012) 120 (21): 481.

https://doi.org/10.1182/blood.V120.21.481.481

Published: 2012

NARROW

TMPRSS6 Antisense Oligonucleotide Therapy Reverses Inflammation in Jak2 V617F Mutant Mice

Combination of a TGF-β Ligand Trap (RAP-GRL) and TMPRSS6 -ASO Is Superior for Correcting β-Thalassemia

Dissecting the Mechanisms of Hepcidin and BMP-SMAD Pathway Regulation By FKBP12

Combination of a Luspatercept-like Drug (RAP-GRL) and Tmprss6 -ASO Is Superior to Either Drug Alone for Correcting β-Thalassemia

Rebalancing Iron Homeostasis and Erythropoiesis through Tfr2 Inhibition for Correction of Anemia in CKD

Correcting β-thalassemia by combined therapies that restrict iron and modulate erythropoietin activity

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

Targeting the BMP Receptor Inhibitor FKBP12 to Upregulate Hepcidin in Wild-Type and Hemojuvelin-Hemochromatosis Models

Correcting Non-Transfusion Dependent β-Thalassemia by Utilizing a Combined Therapy that Modulates EPO Activity by Limiting Erythroid Cellular Iron Intake

Transmembrane Protease, Serine 6 (TMPRSS6) Antisense Oligonucleotide (IONIS-TMPRSS6-L RX ) Reduces Plasma Iron Levels of Healthy Volunteers in a Phase 1 Clinical Study

Inhibiting the Immunophilin FKBP12: A Potential Therapeutic Approach to Iron Overload/Low Hepcidin Disorders

Potential Therapeutic Applications of Jak2 Inhibitors and Hif2a-ASO for the Treatment of β-Thalassemia Intermedia and Major

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

Adult Hemoglobin Production, Chain Rebalance, and Splice Correction in IVS2-745 Beta-Thalassemia Patient Cells Using 2'-O-Methoxyethyl Splice-Switching Oligos

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

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

Novel Targeted Therapy for Precursor B Cell Acute Lymphoblastic Leukemia: Anti-CD22 Antibody-MXD3 Antisense Oligonucleotide Conjugates

Targeting KLF1 for the Treatment of Sickle Cell Disease Using Antisense Oligonucleotides

Targeting BCL11A and KLF1 For The Treatment Of Sickle Cell Disease and β-Thalassemia In Vitro using Antisense Oligonucleotides

Target TMPRSS6 Using Antisense Technology for the Treatment of Hereditary Hemochromatosis and β-Thalassemia

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American Society of Hematology

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American Society of Hematology

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