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

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Year: 2025
Type: conference-abstract
Source: Blood
DOI: 10.1182/blood-2025-4312
Authors: 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
Institutions: University of Rome Tor Vergata, IRCCS Ospedale San Raffaele, The San Raffaele Telethon Institute for Gene Therapy, Fondazione Telethon, Vita-Salute San Raffaele University
Language: English

Topic: Virus-based gene therapy research
Subfield: Genetics
Field: Biochemistry, Genetics and Molecular Biology
Domain: Life Sciences
SDG: Good health and well-being

Open Access status: closed
OpenAlex ID: W4417006129
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