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Long-Term and Real-World Safety and Efficacy of Retroviral Gene Therapy for Adenosine Deaminase Deficiency

Adenosine-deaminase (ADA) deficiency leads to severe combined immunodeficiency (SCID). Previous clinical trials showed that autologous CD34+ cell gene therapy (GT) following busulfan reduced-intensity conditioning is a promising therapeutic approach for ADA-SCID, but long-term data are warranted. He...

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書誌詳細
出版年:Nat Med
主要な著者: Migliavacca, Maddalena, Barzaghi, Federica, Fossati, Claudia, Rancoita, Paola MV, 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
フォーマット: Artigo
言語:Inglês
出版事項: 2024
主題:
オンライン・アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC7615698/
https://ncbi.nlm.nih.gov/pubmed/38355973
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41591-023-02789-4
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