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Journal Articles

Lentiviral hematopoietic stem and progenitor cell gene therapy for Wiskott-Aldrich Syndrome: Early access experience in Italian patients based on 648/1996 law

Free
Sabina Cenciarelli, Stefania Giannelli, Federico Fraschetta, Carmen Caputo, Davide Sala, Ketty Sinigaglia, Mariam Datukishvili, Elena Tomasetto, Francesca Ciotti, Maddalena Fraschini, Federica Salerio, Camilla Ferri, Federica Barzaghi, Valeria Calbi, Daniele Canarutto, Giulia Consiglieri, Matteo Doglio, Francesca Fumagalli, Vera Gallo, Maddalena Migliavacca, Salvatore Recupero, Francesca Tucci, Maria Ester Bernardo, Maria Pia Cicalese, Federica Miotto, Alessandro Aiuti, Francesca Ferrua
Journal: Blood
Blood (2025) 146 (Supplement 1): 4312.
https://doi.org/10.1182/blood-2025-4312
Published: 2025
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

Interleukin-1 blockade in patients with Wiskott-Aldrich syndrome: a retrospective multinational case series

Free
Clinical Trials & Observations Brief Report
Samuele Naviglio, Maria Pia Cicalese, Elizabeth Rivers, Francesca Ferrua, Carmem Bonfim, Sabina Cenciarelli, Kai-Ning Cheong, Maura Faraci, Stefano Giardino, Sujal Ghosh, Pamela P. Lee, Paula Teixeira Lyra, Roland Meisel, Valentina Sofia, Antimo Tessitore, Alberto Tommasini, Erica Valencic, Tanja Christine Vallée, Stefano Volpi, Austen J. Worth, Marco Rabusin, Michael H. Albert, Adrian J. Thrasher, Alessandro Aiuti
Journal: Blood
Blood (2024) 144 (16): 1699–1704.
https://doi.org/10.1182/blood.2024024524
Published: 2024
Includes: Supplemental data
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

Free
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