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Journal Articles

Delivery of Gene Writers In Vivo to Hematopoietic Stem Cells and T Cells Using Targeted and Untargeted Lipid Nanoparticles (LNP)

Michael T. Monte, Lorenzo Tozzi, Rahul Palchaudhuri, Apiwat Wangweerawong, Yaser Heshmati, Giulia Schiroli, Han Gu, Yu Cao, William E. Salomon, Anne Bothmer, Jason Rodriguez, Mohit Gupta, James B. Rottman, William Querbes, Cecilia Cotta-Ramusino, Michael C Holmes, Hari Pujar, Z. Jane Wang

Blood (2024) 144 (Supplement 1): 2197.

https://doi.org/10.1182/blood-2024-208968

Published: 2024

Journal Articles

In Vivo HSC Gene Editing for Correction of the Sickle Cell Mutation Using RNA Gene Writers

Lorenzo Tozzi, Giulia Schiroli, Yaser Heshmati, Yu Cao, Michael T. Monte, Apiwat Wangweerawong, James B. Rottman, Rahul Palchaudhuri, Jean-Antoine Ribeil, John Manis, Hari Pujar, William Querbes, Anne Bothmer, Cecilia Cotta-Ramusino, Z. Jane Wang, William E. Salomon, Michael C Holmes

Blood (2024) 144 (Supplement 1): 515.

https://doi.org/10.1182/blood-2024-208626

Published: 2024

Journal Articles

In Vivo Delivery of RNA By Lipid Nanoparticles (LNP) to Hematopoietic Stem Cells and T Cells in Humanized Mice and Non-Human Primates

Blood (2023) 142 (Supplement 1): 4818.

https://doi.org/10.1182/blood-2023-187271

Published: 2023

Journal Articles

Programmable 3D silk bone marrow niche for platelet generation ex vivo and modeling of megakaryopoiesis pathologies

Christian A. Di Buduo, Lindsay S. Wray, Lorenzo Tozzi, Alessandro Malara, Ying Chen, Chiara E. Ghezzi, Daniel Smoot, Carla Sfara, Antonella Antonelli, Elise Spedden, Giovanna Bruni, Cristian Staii, Luigi De Marco, Mauro Magnani, David L. Kaplan, Alessandra Balduini

Blood (2015) 125 (14): 2254–2264.

https://doi.org/10.1182/blood-2014-08-595561

Published: 2015

Includes: Supplemental data

Journal Articles

Spleen endothelial cells from patients with myelofibrosis harbor the JAK2 V617F mutation

Blood (2013) 121 (2): 360–368.

https://doi.org/10.1182/blood-2012-01-404889

Published: 2013

Includes: Supplemental data

Journal Articles

Combined Inhibition of JAK2 and mTOR Signaling Results in Enhanced Efficacy in in-Vitro and Preclinical Mouse Models of JAK2V617F-Driven Myeloproliferative Disease

Alessandro M. Vannucchi, MD, Niccolò Bartalucci, PhD, Costanza Bogani, PhD, Serena Martinelli, BiSci, Lorenzo Tozzi, PhD, Alberto Bosi, MD, Jean-Luc Villeval, PhD

Blood (2012) 120 (21): 708.

https://doi.org/10.1182/blood.V120.21.708.708

Published: 2012

Journal Articles

Inhibitors of PI3K/Akt and/or mTOR Inhibit the Growth of Cells of Myeloproliferative Neoplasms and Synergize with JAK2 Inhibitor and Interferon,

Alessandro M. Vannucchi, MD, Costanza Bogani, PhD, Niccolò Bartalucci, BiSc, Lorenzo Tozzi, BiSci, Serena Martinelli, BiSci, Paola Guglielmelli, MD, PhD, Chiara Paoli, BiSci, Lisa Pieri, MD, Alberto Bosi, MD

Blood (2011) 118 (21): 3835.

https://doi.org/10.1182/blood.V118.21.3835.3835

Published: 2011

Journal Articles

C-Myb Transactivates the Expression of Erythroid Hsa-miR16-2 Gene,

Elisa Bianchi, PhD, Paola Guglielmelli, MD, Lorenzo Tozzi, BiSci, Costanza Bogani, PhD, Simona Salati, PhD, Roberta Zini, PhD, Ruggiero Norfo, PhD, Valentina Pennucci, PhD, Alessandro M. Vannucchi, M.D., Rossella Manfredini, BiSci, PhD

Blood (2011) 118 (21): 3386.

https://doi.org/10.1182/blood.V118.21.3386.3386

Published: 2011

Journal Articles

Safety and efficacy of everolimus, a mTOR inhibitor, as single agent in a phase 1/2 study in patients with myelofibrosis

Blood (2011) 118 (8): 2069–2076.

https://doi.org/10.1182/blood-2011-01-330563

Published: 2011

Includes: Supplemental data

Journal Articles

Overexpression of microRNA-16-2 contributes to the abnormal erythropoiesis in polycythemia vera

Paola Guglielmelli, Lorenzo Tozzi, Costanza Bogani, Ilaria Iacobucci, Vanessa Ponziani, Giovanni Martinelli, Alberto Bosi, Alessandro M. Vannucchi

Blood (2011) 117 (25): 6923–6927.

https://doi.org/10.1182/blood-2010-09-306506

Published: 2011

Includes: Supplemental data

Journal Articles

A Phase 1/2 Study of RAD001, a mTOR Inhibitor, In Patients with Myelofibrosis: Final Results

Alessandro M. Vannucchi, MD, Paola Guglielmelli, MD, Letizia Lupo, MD, Maria Chiara Finazzi, MD, Lorenzo Tozzi, BiSc, Flavia Biamonte, BiSc, Arianna Masciulli, MD, Maria Chiara Susini, MD, Alessandro Pancrazzi, BiSc, Guido Finazzi, MD, Simona Paratore, MD, Roberto Marchioli, Alberto Bosi, Tiziano Barbui, MD, Alessandro Rambaldi, MD, Giovanni Barosi, MD

Blood (2010) 116 (21): 314.

https://doi.org/10.1182/blood.V116.21.314.314

Published: 2010

Journal Articles

RAD001, An Inhibitor of mTOR, Shows Clinical Activity in a Phase I/II Study in Patients with Primary Myelofibrosis (PMF) and Post Polycythemia Vera/Essential Thrombocythemia Myelofibrosis (PPV/PET MF).

Alessandro M. Vannucchi, MD, Paola Guglielmelli, MD, Elisabetta Gattoni, MD, Elisabetta Antonioli, MD, Costanza Bogani, PhD, Lorenzo Tozzi, BSc, Lisa Pieri, MD, Alberto Bosi, MD, Alessandro Rambaldi, MD, Tiziano Barbui, MD, Giovanni Barosi, MD

Blood (2009) 114 (22): 307.

https://doi.org/10.1182/blood.V114.22.307.307

Published: 2009

Journal Articles

The mTOR Inhibitor, RAD001, Inhibits the Growth of Cells From Patients with Myeloproliferative Neoplasms.

Alessandro M. Vannucchi, MD, Costanza Bogani, PhD, Niccolò Bartalucci, BSc, Paola Guglielmelli, MD, Lorenzo Tozzi, BSc, Elisabetta Antonioli, MD, Lisa Pieri, MD, Alberto Bosi, MD

Blood (2009) 114 (22): 2914.

https://doi.org/10.1182/blood.V114.22.2914.2914

Published: 2009

Journal Articles

Deranged MicroRNA 16-2 Expression Contributes to Erythropoiesis in Polycythemia Vera.

Paola Guglielmelli, MD, Lorenzo Tozzi, BSc, Vanessa Ponziani, PhD, Elisa Bianchi, PhD, Ilaria Iacobucci, PhD, Betty Giusti, PhD, Luciana Rossi, PhD, Giovanni Martinelli, MD, Rossella Manfredini, MD, Alberto Bosi, MD, Alessandro M. Vannucchi, MD

Blood (2009) 114 (22): 3896.

https://doi.org/10.1182/blood.V114.22.3896.3896

Published: 2009

Journal Articles

Dysregulated Expression of MicroRNA-16 Contributes to Abnormal Erythropoiesis in Patients with Polycythemia Vera

Paola Guglielmelli, MD, Lorenzo Tozzi, BiSci, Costanza Bogani, PhD, Niccolo’ Bartalucci, BiSci, Simona Salati, PhD, Rossella Manfredini, MD, Alberto Bosi, MD, Alessandro M. Vannucchi, MD

Blood (2008) 112 (11): 179.

https://doi.org/10.1182/blood.V112.11.179.179

Published: 2008

NARROW

Delivery of Gene Writers In Vivo to Hematopoietic Stem Cells and T Cells Using Targeted and Untargeted Lipid Nanoparticles (LNP)

In Vivo HSC Gene Editing for Correction of the Sickle Cell Mutation Using RNA Gene Writers

In Vivo Delivery of RNA By Lipid Nanoparticles (LNP) to Hematopoietic Stem Cells and T Cells in Humanized Mice and Non-Human Primates

Programmable 3D silk bone marrow niche for platelet generation ex vivo and modeling of megakaryopoiesis pathologies

Spleen endothelial cells from patients with myelofibrosis harbor the JAK2 V617F mutation

Combined Inhibition of JAK2 and mTOR Signaling Results in Enhanced Efficacy in in-Vitro and Preclinical Mouse Models of JAK2V617F-Driven Myeloproliferative Disease

Inhibitors of PI3K/Akt and/or mTOR Inhibit the Growth of Cells of Myeloproliferative Neoplasms and Synergize with JAK2 Inhibitor and Interferon,

C-Myb Transactivates the Expression of Erythroid Hsa-miR16-2 Gene,

Safety and efficacy of everolimus, a mTOR inhibitor, as single agent in a phase 1/2 study in patients with myelofibrosis

Overexpression of microRNA-16-2 contributes to the abnormal erythropoiesis in polycythemia vera

A Phase 1/2 Study of RAD001, a mTOR Inhibitor, In Patients with Myelofibrosis: Final Results

RAD001, An Inhibitor of mTOR, Shows Clinical Activity in a Phase I/II Study in Patients with Primary Myelofibrosis (PMF) and Post Polycythemia Vera/Essential Thrombocythemia Myelofibrosis (PPV/PET MF).

The mTOR Inhibitor, RAD001, Inhibits the Growth of Cells From Patients with Myeloproliferative Neoplasms.

Deranged MicroRNA 16-2 Expression Contributes to Erythropoiesis in Polycythemia Vera.

Dysregulated Expression of MicroRNA-16 Contributes to Abnormal Erythropoiesis in Patients with Polycythemia Vera

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American Society of Hematology

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American Society of Hematology

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