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Paolo Silvani
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Journal Articles

Lentiviral hematopoietic stem and progenitor cell gene therapy with Etuvetidigene autotemcel for the treatment of Wiskott-Aldrich Syndrome

Free
Francesca Ferrua, Sabina Cenciarelli, Stefania Giannelli, Stefania Galimberti, Shanmuganathan Chandrakasan, Federico Fraschetta, Carmen Caputo, Davide Sala, Ilaria Monti, Federica Barzaghi, Valeria Calbi, Daniele Canarutto, Giulia Consiglieri, Matteo Doglio, Vera Gallo, Maddalena Migliavacca, Salvatore Recupero, Francesca Tucci, Alessia Orsini, Raffaella Milani, Mariam Datukishvili, Simona De Gregori, Paolo Silvani, Elena Tomasetto, Koen van Rossem, Federica Miotto, Stefano Zancan, Celeste Scotti, Sean Russell, Luigi Naldini, Fabio Ciceri, Maria Ester Bernardo, Suhag Parikh, Maria Pia Cicalese, Alessandro Aiuti
Journal: Blood
Blood (2025) 146 (Supplement 1): 6092.
https://doi.org/10.1182/blood-2025-6092
Published: 2025
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

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Francesca Ferrua, MD PhD, Maria Pia Cicalese, MD PhD, Stefania Galimberti, PhD, Stefania Giannelli, PhD, Francesca Dionisio, MSc, Federica Barzaghi, MD PhD, Maddalena Migliavacca, MD PhD, Maria Ester Bernardo, MD PhD, Valeria Calbi, MD, Francesca Tucci, MD, Andrea A. Assanelli, MD, Jacopo Peccatori, MD, Elena Albertazzi, PhD, Alessandra G. Clerici, Federica A. Salerio, MSc, Serena Scala, PhD, Luca Basso-Ricci, MSc, Sabina Cenciarelli, MD, Daniele Canarutto, MD, Federico Fraschetta, RN, Antonella Bartoli, MScPharm, Hermann M. Wolf, MD, Paolo Silvani, MD, Salvatore Gattillo, MD, Milena Coppola, MD, Luca Santoleri, MD, Anna Villa, MD, Russell Jones, Chris Dott, PhD, Maria Grazia Valsecchi, PhD, Fabio Ciceri, MD, Luigi Naldini, MD PhD, Alessandro Aiuti, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3346.
https://doi.org/10.1182/blood-2019-124665
Published: 2019
Journal Articles

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

Free
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
Journal: Blood
Blood (2019) 134 (Supplement_1): 607.
https://doi.org/10.1182/blood-2019-128805
Published: 2019