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Journal Articles

Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapy

Blood (2024) 144 (15): 1633–1645.

https://doi.org/10.1182/blood.2023023349

Published: 2024

Includes: Supplemental data

Journal Articles

In Vivo Modification of Hematopoietic Stem Cells By Targeted Lipid Nanoparticles Encapsulating mRNA

Laura Breda, Tyler E Papp, Michael Triebwasser, Amir Yadegari, Megan T Fedorky, Naoto Tanaka, Houping Ni, Drew Weissman, Stefano Rivella, Hamideh Parhiz

Blood (2022) 140 (Supplement 1): 305–306.

https://doi.org/10.1182/blood-2022-165089

Published: 2022

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

Lnp-Targeting Hematopoietic Stem Cells and Lentiviral Gene Transfer to Generate and Rescue a Novel Mouse Model of Lethal Alpha-Thalassemia

Maxwell Chappell, Laura Breda, Danuta Jadwiga Jarocha, Michael Triebwasser, Tyler E Papp, Valentina Ghiaccio, Megan T Fedorky, Amaliris Guerra, Kandace Gollomp, Osheiza Y. Abdulmalik, Nattiya Teawtrakul, Stavros Glentis, Antonis Kattamis, Hamideh Parhiz, Stefano Rivella

Blood (2022) 140 (Supplement 1): 300–302.

https://doi.org/10.1182/blood-2022-166621

Published: 2022

Journal Articles

A Severe Mouse Model of Alpha-Thalassemia to Study Abnormal Iron Metabolism and Erythropoiesis, Hematopoietic Stem Cell Behavior and Development of a Gene Therapy Approach for Its Treatment

Maxwell Chappell, Danuta Jadwiga Jarocha, Laura Breda, Valentina Ghiaccio, Michael Triebwasser, Megan Fedorky, Amaliris Guerra, Kandace Gollomp, Nattiya Teawtrakul, Stavros Glentis, Antonis Kattamis, Osheiza Abdulmalik, Stefano Rivella

Blood (2021) 138 (Supplement 1): 2012.

https://doi.org/10.1182/blood-2021-151217

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

Improved Gene Therapy for Metachromatic Leukodystrophy

Lucas Tricoli, Adeline Vanderver, Laura Adang, Maxwell Chappell, Laura Breda, Naoto Tanaka, Michael Triebwasser, David Wenger, Danuta Jadwiga Jarocha, Amaliris Guerra, Lars Schlotawa, Karthikeyan Radhakrishnan, Rebecca Ahrens-Nicklas, Peter Kurre, Stephanie Hurwitz, Stefano Rivella

Blood (2021) 138 (Supplement 1): 3979.

https://doi.org/10.1182/blood-2021-150986

Published: 2021

Journal Articles

Rescue of Murine IL-7 Receptor Deficiency with Human IL-7 Receptor Gene Therapy

Michael Triebwasser, Danuta Jadwiga Jarocha, Laura Breda, Megan Fedorky, Stefano Rivella

Blood (2021) 138 (Supplement 1): 3131.

https://doi.org/10.1182/blood-2021-151040

Published: 2021

Journal Articles

Elevated P21 (CDKN1a) Mediates Apoptosis of Beta-Thalassemic Erythroid Cells in Mice but Its Ablation Doesn't Improve Erythroid Maturation

Miao Lin, Vijay Menon, PhD, Raymond Liang, PhD, Tasleem Arif, PhD, Laura Breda, PhD, Stefano Rivella, PhD, Saghi Ghaffari, MDPhD

Blood (2020) 136 (Supplement 1): 19.

https://doi.org/10.1182/blood-2020-143384

Published: 2020

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

Lack of Gdf11 does not improve anemia or prevent the activity of RAP-536 in a mouse model of β-thalassemia

Amaliris Guerra, Paraskevi Rea Oikonomidou, Sayantani Sinha, Jianbing Zhang, Vania Lo Presti, Callum R. Hamilton, Laura Breda, Carla Casu, Ping La, Ana C. Martins, Anoop K. Sendamarai, Mark Fleming, Stefano Rivella

Blood (2019) 134 (6): 568–572.

https://doi.org/10.1182/blood.2019001057

Published: 2019

Includes: Supplemental data

Journal Articles

Lack of GDF11 Does Not Ameliorate Erythropoiesis in β-Thalassemia and Does Not Prevent the Activity of the Trap-Ligand RAP-536

Amaliris Guerra, PhD, Rea Oikonomidou, MD, Sinha Gonzalez, PhD, Jianbing Zhang, PhD, Vania Lo Presti, Callum R Hamilton, Laura Breda, PhD, Carla Casu, Mark D. Fleming, MD DPhil, Pedro Martinez, PhD, Rajasekhar Suragani, PhD, Ravi Kumar, PhD, Stefano Rivella

Blood (2018) 132 (Supplement 1): 165.

https://doi.org/10.1182/blood-2018-165

Published: 2018

Journal Articles

AMPK Regulates the Expression of the Fe-S Cluster Assembly Enzyme (ISCU) and ALAS2, Modulating Cellular Iron Metabolism and Increasing Hemoglobin Synthesis

Ping La, Silvia Pires Lourenco, MSc, Laura Breda, PhD, Stefano Rivella

Blood (2018) 132 (Supplement 1): 851.

https://doi.org/10.1182/blood-2018-99-112175

Published: 2018

Journal Articles

Improved Lentiviral Vectors for the Cure of Hemoglobinopathies

Laura Breda, PhD, Valentina Ghiaccio, PhD, Hanyia Zaidi, Silvia Pires Lourenco, MSc, Carla Casu, Kim Smith-Whitley, MD, Janet L. Kwiatkowski, MD, Vanessa Carrion, RN, CPN, Virginia Guzikowski, RN, MSN, CRNP, Ryo Kurita, PhD, Yukio Nakamura, MD PhD, Stefano Rivella, PhD

Blood (2018) 132 (Supplement 1): 3477.

https://doi.org/10.1182/blood-2018-99-111319

Published: 2018

Journal Articles

Generation of New Cell Models to Screen Improved Vectors for Functional Hemoglobin Expression

Silvia Pires Lourenco, MSc, Amaliris Gonzalez, PhD, Tobias Raabe, PhD, Ryo Kurita, PhD, Yukio Nakamura, MD PhD, Laura Breda, PhD, Stefano Rivella, PhD

Blood (2017) 130 (Supplement 1): 5563.

https://doi.org/10.1182/blood.V130.Suppl_1.5563.5563

Published: 2017

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

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

Forced chromatin looping raises fetal hemoglobin in adult sickle cells to higher levels than pharmacologic inducers

Laura Breda, Irene Motta, Silvia Lourenco, Chiara Gemmo, Wulan Deng, Jeremy W. Rupon, Osheiza Y. Abdulmalik, Deepa Manwani, Gerd A. Blobel, Stefano Rivella

Blood (2016) 128 (8): 1139–1143.

https://doi.org/10.1182/blood-2016-01-691089

Published: 2016

Includes: Supplemental data

Journal Articles

Comparing Strategies to Reactivate Fetal Globin Expression for the Treatment of Beta-Globinopathies

Laura Breda, PhD, Jeremy W. Rupon, MD PhD, Irene Motta, MD, Wulan Deng, Gerd A. Blobel, MD PhD, Stefano Rivella, PhD

Blood (2014) 124 (21): 333.

https://doi.org/10.1182/blood.V124.21.333.333

Published: 2014

NARROW

Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapy

In Vivo Modification of Hematopoietic Stem Cells By Targeted Lipid Nanoparticles Encapsulating mRNA

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

Lnp-Targeting Hematopoietic Stem Cells and Lentiviral Gene Transfer to Generate and Rescue a Novel Mouse Model of Lethal Alpha-Thalassemia

A Severe Mouse Model of Alpha-Thalassemia to Study Abnormal Iron Metabolism and Erythropoiesis, Hematopoietic Stem Cell Behavior and Development of a Gene Therapy Approach for Its Treatment

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

Improved Gene Therapy for Metachromatic Leukodystrophy

Rescue of Murine IL-7 Receptor Deficiency with Human IL-7 Receptor Gene Therapy

Elevated P21 (CDKN1a) Mediates Apoptosis of Beta-Thalassemic Erythroid Cells in Mice but Its Ablation Doesn't Improve Erythroid Maturation

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

Lack of Gdf11 does not improve anemia or prevent the activity of RAP-536 in a mouse model of β-thalassemia

Lack of GDF11 Does Not Ameliorate Erythropoiesis in β-Thalassemia and Does Not Prevent the Activity of the Trap-Ligand RAP-536

AMPK Regulates the Expression of the Fe-S Cluster Assembly Enzyme (ISCU) and ALAS2, Modulating Cellular Iron Metabolism and Increasing Hemoglobin Synthesis

Improved Lentiviral Vectors for the Cure of Hemoglobinopathies

Generation of New Cell Models to Screen Improved Vectors for Functional Hemoglobin Expression

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

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

Forced chromatin looping raises fetal hemoglobin in adult sickle cells to higher levels than pharmacologic inducers

Comparing Strategies to Reactivate Fetal Globin Expression for the Treatment of Beta-Globinopathies

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American Society of Hematology

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American Society of Hematology

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