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...

Celý popis

Uloženo v:
Podrobná bibliografie
Vydáno v:Nat Med
Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: 2024
Témata:
Article
On-line přístup: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
Tagy: Přidat tag
Žádné tagy, Buďte první, kdo otaguje tento záznam!

Podobné jednotky