Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency

Migliavacca, Maddalena; Barzaghi, Federica; Fossati, Claudia; Rancoita, Paola M. V.; Gabaldo, Michela; Dionisio, Francesca; Giannelli, Stefania; Salerio, Federica Andrea; Ferrua, Francesca; Tucci, Francesca; Calbi, Valeria; Gallo, Vera; Recupero, Salvatore; Consiglieri, Giulia; Pajno, Roberta; Sambuco, Maria; Priolo, Alessio; Ferri, Chiara; Garella, Vittoria; Monti, Ilaria; Silvani, Paolo; Darin, Silvia; Casiraghi, Miriam; Corti, Ambra; Zancan, Stefano; Levi, Margherita; Cesana, Daniela; Carlucci, Filippo; Pituch-Noworolska, Anna; AbdElaziz, Dalia; Baumann, Ulrich; Finocchi, Andrea; Cancrini, Caterina; Ladogana, Saverio; Meinhardt, Andrea; Meyts, Isabelle; Montin, Davide; Notarangelo, Lucia Dora; Porta, Fulvio; Pasquet, Marlène; Speckmann, Carsten; Stepensky, Polina; Tommasini, Alberto; Rabusin, Marco; Karakas, Zeynep; Galicchio, Miguel; Leonardi, Lucia; Duse, Marzia; Guner, Sukru Nail; Di Serio, Clelia; Ciceri, Fabio; Bernardo, Maria Ester; Aiuti, Alessandro; Cicalese, Maria Pia

doi:10.1038/s41591-023-02789-4

Nat Med

2024

DOI: 10.1038/s41591-023-02789-4

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Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency

Maddalena Migliavacca,

Federica Barzaghi,

Claudia Fossati

et al.

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Cited by 7 publications

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Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency

Cited by 7 publications

References 41 publications

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