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Samantha Scaramuzza
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Journal Articles

Adenine base editor–mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation

Giulia Hardouin, Panagiotis Antoniou, Pierre Martinucci, Tristan Felix, Sandra Manceau, Laure Joseph, Cécile Masson, Samantha Scaramuzza, Giuliana Ferrari, Marina Cavazzana, Annarita Miccio
Journal: Blood
Blood (2023) 141 (10): 1169–1179.
https://doi.org/10.1182/blood.2022016629
Published: 2023
Includes: Supplemental data
Journal Articles

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

Andrea Calabria, Giulio Spinozzi, Daniela Cesana, Fabrizio Benedicenti, Giulia Pais, Serena Scala, Maria Rosa Lidonnici, Samantha Scaramuzza, Alessandra Albertini, Simona Esposito, Fabiola De Mattia, Daniele Canarutto, Francesca Tucci, Maryam Omrani, Francesca Dionisio, Stefania Giannelli, Sarah Marktel, Valeria Calbi, Francesca Ferrua, Bernhard Gentner, Fabio Ciceri, Luigi Naldini, Giuliana Ferrari, Alessandro Aiuti, Eugenio Montini
Journal: Blood
Blood (2022) 140 (Supplement 1): 1904–1905.
https://doi.org/10.1182/blood-2022-162195
Published: 2022
Journal Articles

Hematopoietic Stem Cells Are Endowed with Erythroid Signature in Beta-Thalassemia

Maria Rosa Lidonnici, PhD, Giulia Chianella, Francesca Tiboni, Matteo Barcella, PhD, Ivan Merelli, PhD, Samantha Scaramuzza, PhD, Claudia Rossi, Stefania Crippa, PhD, Maria Ester Bernardo, MD PhD, Fabio Ciceri, MD, Alessandro Aiuti, MD PhD, Sarah Marktel, MD, Giuliana Ferrari, PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 31.
https://doi.org/10.1182/blood-2020-137426
Published: 2020
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

Sarah Marktel, MD, Maria Pia Cicalese, Fabio Giglio, MD, Samantha Scaramuzza, Valeria Calbi, Miriam Casiraghi, Francesca Ciotti, Maria Rosa Lidonnici, Claudia Rossi, Nicoletta Masera, Emanuela D'Angelo, Nadia Mirra, Raffaella Origa, MD, Immacolata Tartaglione, Giacomo Mandelli, Raffaella Milani, Salvatore Gattillo, Milena Coppola, Gianluca Viarengo, Luca Santoleri, Andrea Calabria, Silverio Perrotta, Eugenio Montini, Giovanna Graziadei, MD, Luigi Naldini, MD PhD, Maria Domenica Cappellini, MD, Fabio Ciceri, MD, Alessandro Aiuti, Giuliana Ferrari
Journal: Blood
Blood (2017) 130 (Supplement 1): 355.
https://doi.org/10.1182/blood.V130.Suppl_1.355.355
Published: 2017
Journal Articles

Impairment in the Mesenchymal Bone-Marrow Niche of Beta-Thalassemia Patients and Association with Prolonged Iron Exposure

Stefania Crippa, PhD, Valeria Rossella, Biologist, Laura Silvestri, PhD, Annamaria Aprile, PhD, Samantha Scaramuzza, Silvia Rivis, maria Antonietta Avanzini, Marco Zecca, MD, Sarah Marktel, MD, Fabio Ciceri, MD, Alessandro Aiuti, MD, Giuliana Ferrari, PhD, Maria Ester Bernardo, MD PhD
Journal: Blood
Blood (2017) 130 (Supplement 1): 948.
https://doi.org/10.1182/blood.V130.Suppl_1.948.948
Published: 2017
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
Journal: Blood
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

Francesca Ferrua, MD, Maria Pia Cicalese, MD PhD, Stefania Galimberti, PhD, Samantha Scaramuzza, PhD, Stefania Giannelli, PhD, Roberta Pajno, MD, Francesca Dionisio, PhD, Luca Biasco, PhD, Maria Carmina Castiello, PhD, Miriam Casiraghi, RN, Marcella Facchini, PhD, Andrea Finocchi, MD, Ayse Metin, MD PhD, Jordan S. Orange, MD PhD, Michael H. Albert, MD, Carmen Petrescu, MD, Marita Bosticardo, PhD, Anna Villa, MD, Chris Dott, PhD, Koen van Rossem, MD MSc, PhD, Maria Grazia Valsecchi, PhD, Fabio Ciceri, MD, Maria Grazia Roncarolo, MD, Luigi Naldini, MD PhD, Alessandro Aiuti, MD PhD
Journal: Blood
Blood (2015) 126 (23): 259.
https://doi.org/10.1182/blood.V126.23.259.259
Published: 2015
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
Journal: Blood
Blood (2014) 124 (21): 5.
https://doi.org/10.1182/blood.V124.21.5.5
Published: 2014
Journal Articles

Alterations in the adenosine metabolism and CD39/CD73 adenosinergic machinery cause loss of Treg cell function and autoimmunity in ADA-deficient SCID

Aisha V. Sauer, Immacolata Brigida, Nicola Carriglio, Raisa Jofra Hernandez, Samantha Scaramuzza, Daniela Clavenna, Francesca Sanvito, Pietro L. Poliani, Nicola Gagliani, Filippo Carlucci, Antonella Tabucchi, Maria Grazia Roncarolo, Elisabetta Traggiai, Anna Villa, Alessandro Aiuti
Journal: Blood
Blood (2012) 119 (6): 1428–1439.
https://doi.org/10.1182/blood-2011-07-366781
Published: 2012
Includes: Supplemental data
Journal Articles

Evidence for Efficacy and Safety of Lentiviral Mediated Gene Transfer in T Cells and CD34+ Cells from Wiskott-Aldrich Syndrome Patients.

Samantha Scaramuzza, Sara Trifari, Francesco Marangoni, Silvana Martino, Ayse Metin, Anne Galy, Luigi Naldini, Alessandro Aiuti, Anna Villa, Loic Dupré, Maria Grazia Roncarolo
Journal: Blood
Blood (2006) 108 (11): 3279.
https://doi.org/10.1182/blood.V108.11.3279.3279
Published: 2006
Journal Articles

Long-Term Safety and Efficacy of Stem Cell Gene Therapy for ADA-SCID.

Alessandro Aiuti, Ulrike Benninghoff, Barbara Cassani, Federica Cattaneo, Luciano Callegaro, Grazia Andolfi, Massimiliano Mirolo, Samantha Scaramuzza, Sarah Marktel, Antonella Tabucchi, Filippo Carlucci, Martha Eibl, Memet Aker, Shimon Slavin, Fabio Ciceri, Roberto Miniero, Claudio Bordignon, Maria Grazia Roncarolo
Journal: Blood
Blood (2006) 108 (11): 200.
https://doi.org/10.1182/blood.V108.11.200.200
Published: 2006
Journal Articles

Clonal Analyses of Retroviral Vector Integrations in ADA-SCID Patients Treated with Stem Cell Gene Therapy.

Barbara Cassani, Grazia Andolfi, Massimiliano Mirolo, Luca Biasco, Alessandra Recchia, Fabrizia Urbinati, Cristina Valacca, Samantha Scaramuzza, Matteo Cazzola, Daniela Sartori, Alessandro Ambrosi, Clelia Di Serio, Maria Grazia Roncarolo, Fulvio Mavilio, Claudio Bordignon, Alessandro Aiuti
Journal: Blood
Blood (2006) 108 (11): 3249.
https://doi.org/10.1182/blood.V108.11.3249.3249
Published: 2006
Journal Articles

Functional Correction of Lymphocytes and Dendritic Cells of Patients with WAS or XLT, Using Lentiviral Vectors Targeting the Expression of WAS Protein to Hematopoietic Cells.

Sabine Charrier, PhD, Loic Dupre, PhD, Mike Blundell, PhD, Samantha Scaramuzza, Laurence Jeanson, PhD, William Vainchenker, MD, Olivier Danos, PhD, Maria-Grazia Roncarolo, MD, Adrian Thrasher, MD, Anne Galy, PhD
Journal: Blood
Blood (2005) 106 (11): 3042.
https://doi.org/10.1182/blood.V106.11.3042.3042
Published: 2005