换一批Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency.
第一作者：
Maddalena,Migliavacca
第一单位：
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.;Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
作者：
Maddalena,Migliavacca [1]
;
Federica,Barzaghi [1]
;
Claudia,Fossati [2]
;
Paola M V,Rancoita [3]
;
Michela,Gabaldo [4]
;
Francesca,Dionisio [2]
;
Stefania,Giannelli [2]
;
Federica Andrea,Salerio [2]
;
Francesca,Ferrua [1]
;
Francesca,Tucci [1]
;
Valeria,Calbi [1]
;
Vera,Gallo [1]
;
Salvatore,Recupero [1]
;
Giulia,Consiglieri [1]
;
Roberta,Pajno [1]
;
Maria,Sambuco [1]
;
Alessio,Priolo [1]
;
Chiara,Ferri [5]
;
Vittoria,Garella [5]
;
Ilaria,Monti [2]
;
Paolo,Silvani [6]
;
Silvia,Darin [2]
;
Miriam,Casiraghi [2]
;
Ambra,Corti [2]
;
Stefano,Zancan [4]
;
Margherita,Levi [4]
;
Daniela,Cesana [2]
;
Filippo,Carlucci [7]
;
Anna,Pituch-Noworolska [8]
;
Dalia,AbdElaziz [9]
;
Ulrich,Baumann [10]
;
Andrea,Finocchi [11]
;
Caterina,Cancrini [11]
;
Saverio,Ladogana [12]
;
Andrea,Meinhardt [13]
;
Isabelle,Meyts [14]
;
Davide,Montin [15]
;
Lucia Dora,Notarangelo [16]
;
Fulvio,Porta [17]
;
Marlène,Pasquet [18]
;
Carsten,Speckmann [19]
;
Polina,Stepensky [20]
;
Alberto,Tommasini [21]
;
Marco,Rabusin [21]
;
Zeynep,Karakas [22]
;
Miguel,Galicchio [23]
;
Lucia,Leonardi [24]
;
Marzia,Duse [24]
;
Sukru Nail,Guner [25]
;
Clelia,Di Serio [26]
;
Fabio,Ciceri [27]
;
Maria Ester,Bernardo [28]
;
Alessandro,Aiuti [29]
;
Maria Pia,Cicalese [28]
作者单位：
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.;Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
[1]
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
[2]
University Centre for Statistics in the Biomedical Sciences (CUSSB), Vita-Salute San Raffaele University, Milan, Italy.
[3]
Fondazione Telethon, Milan, Italy.
[4]
Università Vita-Salute San Raffaele, Milan, Italy.
[5]
Department of Anesthesia and Critical Care, IRCCS San Raffaele Scientific Institute, Milan, Italy.
[6]
Department of Medical Biotechnologies, University of Siena, Siena, Italy.
[7]
Department of Immunology, University Children's Hospital of Cracow, Cracow, Poland.
[8]
Department of Pediatrics, Faculty of Medicine, Cairo University, Cairo, Egypt.
[9]
Department of Paediatric Pulmonology, Allergy and Neonatology, Hannover Medical School, Hannover, Germany.
[10]
Research Unit of Primary Immunodeficiencies, Academic Department of Pediatrics, Bambino Gesù Children's Hospital, Scientific Institute for Research and Healthcare (IRCCS), Rome, Italy.;Department of Systems Medicine, University of Rome Tor Vergata, Rome, Italy.
[11]
Paediatric Onco-haematology Unit, 'Casa Sollievo della Sofferenza' Hospital, IRCCS, San Giovanni Rotondo, Italy.
[12]
Department of Pediatric Hematology and Oncology, Medical Center, University Hospital Giessen, Giessen, Germany.
[13]
Laboratory of Inborn Errors of Immunity, Department of Microbiology, Immunology and Transplantation, KU Leuven, Childhood Immunology, Department of Pediatrics, UZ Leuven, Leuven, Belgium.
[14]
Department of Pediatric and Public Health Sciences, University of Torino, Turin, Italy.;Regina Margherita Children's Hospital, AOU Città della Salute e della Scienza di Torino, Turin, Italy.
[15]
Medical Direction, Children's Hospital, ASST-Spedali Civili, Brescia, Italy.
[16]
Pediatric Oncology-Hematology and BMT Unit, Spedali Civili di Brescia, Brescia, Italy.
[17]
Pediatric Hematology and Immunology, Children's Hospital, Toulouse, France.
[18]
Institute for Immunodeficiency, Center for Chronic Immunodeficiency (CCI), Faculty of Medicine, Medical Center, University of Freiburg, Freiburg, Germany.;Division of Pediatric Hematology and Oncology, Department of Pediatric and Adolescent Medicine, University Medical Center Freiburg, University of Freiburg, Freiburg, Germany.
[19]
Department of Bone Marrow Transplantation and Cancer Immunotherapy and Faculty of Medicine, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.
[20]
Department of Pediatrics, Institute for Maternal and Child Health, IRCCS Burlo Garofolo, Trieste, Italy.
[21]
Department of Pediatrics, Hematology/Oncology Unit, Istanbul School of Medicine, Istanbul University, Istanbul, Turkey.
[22]
Allergy and Immnunology Service, Hospital de Niños VJ Vilela, Rosario, Argentina.
[23]
Department of Maternal, Infantile and Urological Sciences, Sapienza University of Rome, Rome, Italy.
[24]
Division of Pediatric Allergy and Immunology, Faculty of Medicine, Necmettin Erbakan University, Konya, Turkey.
[25]
University Centre for Statistics in the Biomedical Sciences (CUSSB), Vita-Salute San Raffaele University, Milan, Italy.;Faculty of Biomedical Sciences, Università della Svizzera Italiana, Lugano, Switzerland.
[26]
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.;Università Vita-Salute San Raffaele, Milan, Italy.;Hematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
[27]
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.;Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.;Università Vita-Salute San Raffaele, Milan, Italy.
[28]
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy. aiuti.alessandro@hsr.it.;Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy. aiuti.alessandro@hsr.it.;Università Vita-Salute San Raffaele, Milan, Italy. aiuti.alessandro@hsr.it.
[29]
医学主题词
人类(Humans);重症联合免疫缺陷(Severe Combined Immunodeficiency);腺苷脱氨酶(Adenosine Deaminase);白消安(Busulfan);逆转录病毒科(Retroviridae);造血干细胞移植(Hematopoietic Stem Cell Transplantation);丙种球蛋白缺乏血症(Agammaglobulinemia)
DOI
10.1038/s41591-023-02789-4
PMID
38355973
发布时间
2025-12-02
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Nature medicine
Nature medicine
488-497页
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