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Journal Articles

Real-world comparative effects of curative and disease-modifying therapies on ineffective erythropoiesis in beta-thalassemia

Carlo Castruccio Castracani, Madison Bright, Jaladhikumar Patel, Scott Peslak, Aaron Cheng, Laura Breda, Stefano Rivella, Janet Kwiatkowski

Blood (2025) 146 (Supplement 1): 176.

https://doi.org/10.1182/blood-2025-176

Published: 2025

Journal Articles

Expansion of human hematopoietic stem cells by inhibiting translation

Chenchen Li, Hanna Shin, Dheeraj Bhavanasi, Xiaolei Liu, Mai Liu, Yu Xiang, Scott Peslak, Friederike Herbst, Mohamad-Gabriel Alameh, Laura Breda, Stefano Rivella, Juan Alvarez-Dominguez, Gerd Blobel, Brian Gregory, Jian Huang, Peter Klein

Blood (2025) 146 (Supplement 1): 1379.

https://doi.org/10.1182/blood-2025-1379

Published: 2025

Journal Articles

Norad long noncoding RNA, a decoy for pumilio RNA-binding proteins, elevates fetal hemoglobin levels

Anita Dhara, Parina Patel, Rachael White, Sarah Adams, Raja Sundari Sundaram, Chiara Federici, Sumayea Mahmood Meem, Ada Chang, Carlo Castruccio Castracani, Stefano Rivella, Jane Little, Umut Gurkan, Mahesh Ramamoorthy, Merlin Nithya Gnanapragasam

Blood (2025) 146 (Supplement 1): 610.

https://doi.org/10.1182/blood-2025-610

Published: 2025

Journal Articles

TNFα signaling restores steady-state hematopoiesis in a TNFαKO mouse model of anemia of inflammation

Amaliris Guerra, Vania Lo Presti, Ding-Wen Chen, Ana Catarina Martins, Ariel Rivera, Nolan Hamilton, Pankja Sharma, Yelena Z. Ginzburg, Carlo Castruccio Castracani, Carla Casu, Ritama Gupta, Raffaella Gozzelino, Edward M. Behrens, Laura F. Bennett, Robert F. Paulson, Peter Kurre, Stefano Rivella

Blood (2025) 146 (8): 984–997.

https://doi.org/10.1182/blood.2025029352

Published: 2025

Includes: Supplemental data

Journal Articles

An erythroid-specific lentiviral vector improves anemia and iron metabolism in a new model of XLSA

Carlo Castruccio Castracani, Laura Breda, Tyler E. Papp, Amaliris Guerra, Enrico Radaelli, Charles-Antoine Assenmacher, Giovanni Finesso, Barbara L. Mui, Ying K. Tam, Simona Fontana, Chiara Riganti, Veronica Fiorito, Sara Petrillo, Emanuela Tolosano, Hamideh Parhiz, Stefano Rivella

Blood (2025) 145 (1): 98–113.

https://doi.org/10.1182/blood.2024025846

Published: 2025

Includes: Supplemental data

Journal Articles

TFR2 Coordinates Erythroid Iron Availability and Erythropoietin Receptor Sensitivity By Monoferric Transferrin Forms

Amaliris Guerra, Pankaj Sharma, Xiuli An, Nermi L. Parrow, Robert E. Fleming, Yelena Ginzburg, Stefano Rivella

Blood (2024) 144 (Supplement 1): 2468.

https://doi.org/10.1182/blood-2024-211984

Published: 2024

Journal Articles

In Utero mRNA Lipid Nanoparticle Delivery in a Murine Model of Fanconi Anemia

Linah Omer, William Johnston, Sarah Adams, Omar Banda, Elena Sofia Vivona, Stefano Rivella, Mohamad-Gabriel Alameh, William Peranteau, Peter Kurre

Blood (2024) 144 (Supplement 1): 2202.

https://doi.org/10.1182/blood-2024-211241

Published: 2024

Journal Articles

An Erythroid-Specific Lentiviral Vector Improves Anemia and Iron Metabolism in a New Model of Xlsa

Carlo Castruccio Castracani, Laura Breda, Tyler E. Papp, Amaliris Guerra, Enrico Radaelli, Charles-Antoine Assenmacher, Giovanni Finesso, Barbara Mui, Ying K. Tam, Simona Fontana, Chiara Riganti, Veronica Fiorito, Sara Petrillo, Emanuela Tolosano, Hamideh Parhiz, Stefano Rivella

Blood (2024) 144 (Supplement 1): 2195.

https://doi.org/10.1182/blood-2024-200747

Published: 2024

Journal Articles

TNFα Plays Essential Role in Resolution of Anemia of Inflammation in Tnfαko Mice Treated with Heat-Killed Brucella Abortus

Vania Lo Presti, Ding-wen Chen, Ana Martins, Pankaj Sharma, Yelena Ginzburg, Carlo Castruccio Castracani, Carla Casu, Ritama Gupta, Raffaella Gozzelino, Edward Behrens, Laura F Bennett, Robert Paulson, Peter Kurre, Stefano Rivella, Amaliris Guerra

Blood (2024) 144 (Supplement 1): 168.

https://doi.org/10.1182/blood-2024-212272

Published: 2024

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

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

Differential Effects of Monoferric Transferrin Forms on Erythropoiesis Are Mediated By TFR2

Amaliris Guerra, Ariel Rivera, Nolan Hamilton, Xiuli An, Nermi L. Parrow, Yelena Ginzburg, Robert E. Fleming, Stefano Rivella

Blood (2023) 142 (Supplement 1): 3846.

https://doi.org/10.1182/blood-2023-189107

Published: 2023

Journal Articles

Differential Effect of Transferrin Lobe Iron Occupancy on a Murine Model of Inflammation

Nermi L. Parrow, Nisha Ajit George, Faris Ali, Nadia Wattad, Amaliris Guerra, Yelena Ginzburg, Stefano Rivella, Robert E. Fleming

Blood (2023) 142 (Supplement 1): 1095.

https://doi.org/10.1182/blood-2023-190984

Published: 2023

Journal Articles

Phenotypic HSPC Rescue By RNA Lipid Nanoparticles in a Murine Model of Fanconi Anemia

Linah Omer, Omar Banda, Sarah Adams, Seul K Jung, Stefano Rivella, Mohamad-Gabriel Alameh, Drew Weissman, Peter Kurre

Blood (2023) 142 (Supplement 1): 3625.

https://doi.org/10.1182/blood-2023-188193

Published: 2023

Journal Articles

Differential Effects of Monoferric Forms of Transferrin on Mouse Models of Anemia of Inflammation and Congenital Anemia

Amaliris Guerra, Ana Catarina Martins, Ariel Rivera, Nermi Parrow, Raffaella Gozzelino, Yelena Ginzburg, Robert E Fleming, Stefano Rivella

Blood (2022) 140 (Supplement 1): 8179–8180.

https://doi.org/10.1182/blood-2022-166391

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

Establishment of High Titer Baboon Endogenous Retrovirus Envelope Pseudotyped Lentiviral Vector Production Protocol Focusing on Syncytium Formation and Exfoliation

Kazuhiro Noguchi, Yasuhiro Ikawa, Mika Takenaka, Yuta Sakai, Toshihiro Fujiki, Rie Kuroda, Maxwell Chappell, Valentina Ghiaccio, Stefano Rivella, Taizo Wada

Blood (2022) 140 (Supplement 1): 12987–12989.

https://doi.org/10.1182/blood-2022-163144

Published: 2022

Journal Articles

TNFα and Anti-IFNγ Rescues Chronic Anemia of Inflammation Phenotype in Tnfαko Mice Treated with Heat-Killed Brucella Abortus

Amaliris Guerra, Vania Lo Presti, Ding-Wen Chen, Ana Catarina Martins, Carlo Castruccio Castracani, Ritama Gupta, Laura Bennett, Raffaella Gozzelino, Robert Paulson, Peter Kurre, Stefano Rivella, Carla Casu

Blood (2022) 140 (Supplement 1): 5302–5303.

https://doi.org/10.1182/blood-2022-166643

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

Direct Delivery of mRNA-Loaded Lipid Nanoparticles to the Hematopoietic Stem Cell Niche

Omar Banda, Mohamad-Gabriel Alameh, Seul Jung, Drew Weissman, Stefano Rivella, Peter Kurre

Blood (2022) 140 (Supplement 1): 4923–4924.

https://doi.org/10.1182/blood-2022-159085

Published: 2022

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Real-world comparative effects of curative and disease-modifying therapies on ineffective erythropoiesis in beta-thalassemia

Expansion of human hematopoietic stem cells by inhibiting translation

Norad long noncoding RNA, a decoy for pumilio RNA-binding proteins, elevates fetal hemoglobin levels

TNFα signaling restores steady-state hematopoiesis in a TNFαKO mouse model of anemia of inflammation

An erythroid-specific lentiviral vector improves anemia and iron metabolism in a new model of XLSA

TFR2 Coordinates Erythroid Iron Availability and Erythropoietin Receptor Sensitivity By Monoferric Transferrin Forms

In Utero mRNA Lipid Nanoparticle Delivery in a Murine Model of Fanconi Anemia

An Erythroid-Specific Lentiviral Vector Improves Anemia and Iron Metabolism in a New Model of Xlsa

TNFα Plays Essential Role in Resolution of Anemia of Inflammation in Tnfαko Mice Treated with Heat-Killed Brucella Abortus

TMPRSS6 Antisense Oligonucleotide Therapy Reverses Inflammation in Jak2 V617F Mutant Mice

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

Differential Effects of Monoferric Transferrin Forms on Erythropoiesis Are Mediated By TFR2

Differential Effect of Transferrin Lobe Iron Occupancy on a Murine Model of Inflammation

Phenotypic HSPC Rescue By RNA Lipid Nanoparticles in a Murine Model of Fanconi Anemia

Differential Effects of Monoferric Forms of Transferrin on Mouse Models of Anemia of Inflammation and Congenital Anemia

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

Establishment of High Titer Baboon Endogenous Retrovirus Envelope Pseudotyped Lentiviral Vector Production Protocol Focusing on Syncytium Formation and Exfoliation

TNFα and Anti-IFNγ Rescues Chronic Anemia of Inflammation Phenotype in Tnfαko Mice Treated with Heat-Killed Brucella Abortus

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

Direct Delivery of mRNA-Loaded Lipid Nanoparticles to the Hematopoietic Stem Cell Niche

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American Society of Hematology

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