Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency-Reference-Cited by-同舟云学术

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

Published:2024-02 Issue:2 Volume:30 Page:488-497
ISSN:1078-8956
Container-title:Nature Medicine
language:en
Short-container-title:Nat Med

Author:

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^ORCID,Gallo Vera,Recupero Salvatore,Consiglieri Giulia,Pajno Roberta,Sambuco Maria,Priolo Alessio^ORCID,Ferri Chiara,Garella Vittoria,Monti Ilaria,Silvani Paolo,Darin Silvia,Casiraghi Miriam,Corti Ambra,Zancan Stefano,Levi Margherita,Cesana Daniela^ORCID,Carlucci Filippo,Pituch-Noworolska Anna,AbdElaziz Dalia^ORCID,Baumann Ulrich^ORCID,Finocchi Andrea,Cancrini Caterina,Ladogana Saverio,Meinhardt Andrea^ORCID,Meyts Isabelle^ORCID,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^ORCID,Guner Sukru Nail^ORCID,Di Serio Clelia,Ciceri Fabio^ORCID,Bernardo Maria Ester,Aiuti Alessandro^ORCID,Cicalese Maria Pia

Funder

Fondazione Telethon

Else Kröner-Fresenius-Stiftung

Publisher

Springer Science and Business Media LLC

Link

https://www.nature.com/articles/s41591-023-02789-4.pdf

Reference42 articles.

1. Hassan, A. et al. Inborn Errors Working Party of the European Group for Blood and Marrow Transplantation and European Society for Immunodeficiency. Outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency. Blood 120, 3615–3624 (2012).

2. Kreins, A. Y. et al. Long-term immune recovery after hematopoietic stem cell transplantation for ADA deficiency: a single-center experience. J. Clin. Immunol. 42, 94–107 (2022).

3. Ghimenton, E. et al. Hematopoietic cell transplantation for adenosine deaminase severe combined immunodeficiency—improved outcomes in the modern era. J. Clin. Immunol. 42, 819–826 (2022).

4. Lankester, A. et al. Hematopoietic cell transplantation in severe combined immunodeficiency: the SCETIDE 2006-2014 European cohort. J. Allergy Clin. Immunol. 149, 1744–1754 (2022).

5. Geoffrey, D. E. et al. Outcomes following treatment for adenosine deaminase deficient severe combined immunodeficiency: a report from the PIDTC. Blood 140, 685–705 (2022).

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