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Journal Articles

Unraveling Pathophysiology and Hematopoiesis of Vexas Syndrome By Multi-Omics Analyses and Targeted Gene Editing

Blood (2023) 142 (Supplement 1): 2692.

https://doi.org/10.1182/blood-2023-179749

Published: 2023

Journal Articles

Genetic Engineering of Hematopoietic Progenitor Stem Cells for Targeted IFN-α Immunotherapy Reprogramming the Solid Tumor Microenvironment: A First-in-Man Study in Glioblastoma Multiforme (NCT03866109)

Blood (2023) 142 (Supplement 1): 4850.

https://doi.org/10.1182/blood-2023-178212

Published: 2023

Journal Articles

Lipid nanoparticles allow efficient and harmless ex vivo gene editing of human hematopoietic cells

Valentina Vavassori, Samuele Ferrari, Stefano Beretta, Claudia Asperti, Luisa Albano, Andrea Annoni, Chiara Gaddoni, Angelica Varesi, Monica Soldi, Alessandro Cuomo, Tiziana Bonaldi, Marina Radrizzani, Ivan Merelli, Luigi Naldini

Blood (2023) 142 (9): 812–826.

https://doi.org/10.1182/blood.2022019333

Published: 2023

Includes: Supplemental data

Journal Articles

Adaptive Routes of Hematopoietic Stem Cell Differentiation to Disease Conditions and Age in Gene Therapy Patients

Blood (2022) 140 (Supplement 1): 1904–1905.

https://doi.org/10.1182/blood-2022-162195

Published: 2022

Journal Articles

Reprogramming Macrophages Using Autologous Hematopoietic Stem Cells As Immunotherapy for Glioblastoma: TEM-GBM Study (NCT03866109)

Blood (2022) 140 (Supplement 1): 4575–4576.

https://doi.org/10.1182/blood-2022-165975

Published: 2022

Journal Articles

Assessing Stealth and Sensed Base Editing in Human Hematopoietic Stem/Progenitor Cells

Martina Fiumara, Samuele Ferrari, Attya Omer, Stefano Beretta, Luisa Albano, Ivan Merelli, Luigi Naldini

Blood (2021) 138 (Supplement 1): 3976.

https://doi.org/10.1182/blood-2021-147700

Published: 2021

Journal Articles

Towards Clinical Translation of Hematopoietic Cell Gene Editing for Treating Hyper-IgM Type 1

Valentina Vavassori, Elisabetta Mercuri, Genni Marcovecchio, Maria Carmina Castiello, Daniele Canarutto, Claudia Asperti, Aurelien Jacob, Luisa Albano, Elena Fontana, Eugenio Scanziani, Marina Radrizzani, Anna Villa, Pietro Genovese, Luigi Naldini

Blood (2021) 138 (Supplement 1): 3978.

https://doi.org/10.1182/blood-2021-148572

Published: 2021

Journal Articles

TEM-GBM: An Open-Label, Phase I/IIa Dose-Escalation Study Evaluating the Safety and Efficacy of Genetically Modified Tie-2 Expressing Monocytes to Deliver IFN-α within Glioblastoma Tumor Microenvironment

Blood (2021) 138 (Supplement 1): 2844.

https://doi.org/10.1182/blood-2021-150230

Published: 2021

Journal Articles

Lentiviral-Mediated Gene Therapy for the Treatment of Adenosine Deaminase 2 Deficiency

Alessandra Mortellaro, Matteo Zoccolillo, Cristina Mesa Nuñez, Alessia Brix, Immacolata Brigida, Federica Barzaghi, Serena Scala, Raisa Jofra Hernández, Luca Basso-Ricci, Mariasilvia Colantuoni, Simone Cesaro, Francesca Conti, Andrea Pession, Fabio Benedetti, Marco Gattorno, Luigi Naldini, Maria Pia Cicalese, Anna Pistocchi, Alessandro Aiuti

Blood (2021) 138 (Supplement 1): 2937.

https://doi.org/10.1182/blood-2021-152127

Published: 2021

Journal Articles

Extensive Metabolic Correction of Hurler Disease By Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II Trial

Bernhard Gentner, MD, Maria Ester Bernardo, MD PhD, Francesca Tucci, MD, Erika Zonari, PhD, Francesca Fumagalli, MD, Silvia Pontesilli, MD, Serena Acquati, PhD, Paolo Silvani, MD, Fabio Ciceri, MD, Attilio Rovelli, MD, Giancarlo La Marca, PhD, Rossella Parini, MD, Silvia Gregori, PhD, Eugenio Montini, PhD, Luigi Naldini, MD PhD, Alessandro Aiuti, MD PhD

Blood (2019) 134 (Supplement_1): 607.

https://doi.org/10.1182/blood-2019-128805

Published: 2019

Journal Articles

TEM-MM-101: A Phase I/IIa Dose Escalation Study Evaluating the Safety and Activity of Autologous CD34+ Enriched Hematopoietic Progenitor Cells Genetically Modified for Human Interferon-α2 in Multiple Myeloma Patients with Early Relapse after Intensive Front Line Therapy

Bernhard Gentner, MD, Francesca Farina, MD, Matteo Giovanni Carrabba, MD, Magda Marcatti, MD, Stefania Girlanda, MD, Gabriele Antonarelli, MD, Capotondo Alessia, PhD, Stefania Mazzoleni, PhD, Carlo Russo, MD, Luigi Naldini, MD PhD, Fabio Ciceri, MD

Blood (2019) 134 (Supplement_1): 2064.

https://doi.org/10.1182/blood-2019-123749

Published: 2019

Journal Articles

Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS): Up to 8 Years of Follow up in 17 Subjects Treated Since 2010

Blood (2019) 134 (Supplement_1): 3346.

https://doi.org/10.1182/blood-2019-124665

Published: 2019

Journal Articles

Generation of Memory Stem T Cells Specific for Tumor Antigens and Resistant to Inhibitory Signals By Genome Editing

Beatrice Claudia Cianciotti, Alessia Potenza, Valentina Vavassori, Zulma Magnani, Luigi Naldini, MD PhD, Fabio Ciceri, MD, Pietro Genovese, PhD, Eliana Ruggiero, PhD, Chiara Bonini, MD

Blood (2018) 132 (Supplement 1): 2202.

https://doi.org/10.1182/blood-2018-99-118201

Published: 2018

Journal Articles

Liver-Directed Gene Therapy for Hemophilia B with Immune Stealth Lentiviral Vectors

Alessio Cantore, Michela Milani, Andrea Annoni, Tongyao Liu, Sara Bartolaccini, Mauro Biffi, Fabio Russo, Robert Peters, PhD, Angelo Lombardo, Timothy C. Nichols, MD, Eduard Ayuso, Luigi Naldini

Blood (2017) 130 (Supplement 1): 605.

https://doi.org/10.1182/blood.V130.Suppl_1.605.605

Published: 2017

Journal Articles

Gene Therapy for Beta Thalassemia: Preliminary Results from the PHASE I/II Tiget-Bthal Trial of Autologous Hematopoietic Stem Cells Genetically Modified with GLOBE Lentiviral Vector

Blood (2017) 130 (Supplement 1): 355.

https://doi.org/10.1182/blood.V130.Suppl_1.355.355

Published: 2017

Journal Articles

NY-ESO-1 TCR single edited stem and central memory T cells to treat multiple myeloma without graft-versus-host disease

Blood (2017) 130 (5): 606–618.

https://doi.org/10.1182/blood-2016-08-732636

Published: 2017

Includes: Supplemental data

Journal Articles

Incremental Innovation of Ex Vivo Hematopoietic Stem Cell Engineering to Expand Clinical Gene Therapy Applications

Erika Zonari, PhD, Giacomo Desantis, PhD, Carolina Petrillo, Oriana Meo, Samantha Scaramuzza, PhD, Maria Rosa Lidonnici, PhD, Anna Kajaste-Rudnitski, PhD, Fabio Ciceri, MD, Sarah Marktel, Alessandro Aiuti, MD PhD, Giuliana Ferrari, PhD, Luigi Naldini, MD PhD, Bernhard Gentner, MD

Blood (2016) 128 (22): 4707.

https://doi.org/10.1182/blood.V128.22.4707.4707

Published: 2016

Journal Articles

Safety and Clinical Benefit of Lentiviral Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Blood (2015) 126 (23): 259.

https://doi.org/10.1182/blood.V126.23.259.259

Published: 2015

Journal Articles

NY-ESO-1 Single Edited T Cells to Treat Multiple Myeloma without Inducing GvHD

Sara Mastaglio, MD, Pietro Genovese, PhD, Zulma Magnani, MSc, Elisa Landoni, Barbara Camisa, BSc, Giulia Schiroli, Elena Provasi, PhD, Angelo Lombardo, PhD, Andreas Reik, PhD, Nicoletta Cieri, MD PhD, Maurilio Ponzoni, Fabio Ciceri, MD, Claudio Bordignon, MD, Michael C. Holmes, PhD, Philip D Gregory, PhD, Luigi Naldini, MD PhD, Chiara Bonini, MD

Blood (2014) 124 (21): 308.

https://doi.org/10.1182/blood.V124.21.308.308

Published: 2014

Journal Articles

Comprehensive Clonal Mapping of Hematopoiesis in Vivo in Humans By Retroviral Vector Insertional Barcoding

Luca Biasco, PhD, Serena Scala, Francesca Dionisio, Andrea Calabria, Luca Basso Ricci, Samantha Scaramuzza, Cristina Baricordi, Stefania Giannelli, Victor X Neduva, David J Dow, Danilo Pellin, Paola Vicard, Clelia Di Serio, Eugenio Montini, Luigi Naldini, Alessandro Aiuti

Blood (2014) 124 (21): 5.

https://doi.org/10.1182/blood.V124.21.5.5

Published: 2014

NARROW

Unraveling Pathophysiology and Hematopoiesis of Vexas Syndrome By Multi-Omics Analyses and Targeted Gene Editing

Genetic Engineering of Hematopoietic Progenitor Stem Cells for Targeted IFN-α Immunotherapy Reprogramming the Solid Tumor Microenvironment: A First-in-Man Study in Glioblastoma Multiforme (NCT03866109)

Lipid nanoparticles allow efficient and harmless ex vivo gene editing of human hematopoietic cells

Adaptive Routes of Hematopoietic Stem Cell Differentiation to Disease Conditions and Age in Gene Therapy Patients

Reprogramming Macrophages Using Autologous Hematopoietic Stem Cells As Immunotherapy for Glioblastoma: TEM-GBM Study (NCT03866109)

Assessing Stealth and Sensed Base Editing in Human Hematopoietic Stem/Progenitor Cells

Towards Clinical Translation of Hematopoietic Cell Gene Editing for Treating Hyper-IgM Type 1

TEM-GBM: An Open-Label, Phase I/IIa Dose-Escalation Study Evaluating the Safety and Efficacy of Genetically Modified Tie-2 Expressing Monocytes to Deliver IFN-α within Glioblastoma Tumor Microenvironment

Lentiviral-Mediated Gene Therapy for the Treatment of Adenosine Deaminase 2 Deficiency

Extensive Metabolic Correction of Hurler Disease By Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II Trial

TEM-MM-101: A Phase I/IIa Dose Escalation Study Evaluating the Safety and Activity of Autologous CD34+ Enriched Hematopoietic Progenitor Cells Genetically Modified for Human Interferon-α2 in Multiple Myeloma Patients with Early Relapse after Intensive Front Line Therapy

Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS): Up to 8 Years of Follow up in 17 Subjects Treated Since 2010

Generation of Memory Stem T Cells Specific for Tumor Antigens and Resistant to Inhibitory Signals By Genome Editing

Liver-Directed Gene Therapy for Hemophilia B with Immune Stealth Lentiviral Vectors

Gene Therapy for Beta Thalassemia: Preliminary Results from the PHASE I/II Tiget-Bthal Trial of Autologous Hematopoietic Stem Cells Genetically Modified with GLOBE Lentiviral Vector

NY-ESO-1 TCR single edited stem and central memory T cells to treat multiple myeloma without graft-versus-host disease

Incremental Innovation of Ex Vivo Hematopoietic Stem Cell Engineering to Expand Clinical Gene Therapy Applications

Safety and Clinical Benefit of Lentiviral Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

NY-ESO-1 Single Edited T Cells to Treat Multiple Myeloma without Inducing GvHD

Comprehensive Clonal Mapping of Hematopoiesis in Vivo in Humans By Retroviral Vector Insertional Barcoding

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American Society of Hematology

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American Society of Hematology

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