B-cell development and functions and therapeutic options in adenosine deaminase-deficient patients.

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第一作者： Immacolata,Brigida

第一单位： San Raffaele Telethon Institute for Gene Therapy (TIGET), San Raffaele Scientific Institute, Milan, Italy.

作者： Immacolata,Brigida ^[1] ; Aisha V,Sauer ^[1] ; Francesca,Ferrua ^[2] ; Stefania,Giannelli ^[1] ; Samantha,Scaramuzza ^[1] ; Valentina,Pistoia ^[1] ; Maria Carmina,Castiello ^[3] ; Barbara H,Barendregt ^[4] ; Maria Pia,Cicalese ^[2] ; Miriam,Casiraghi ^[2] ; Chiara,Brombin ^[5] ; Jennifer,Puck ^[6] ; Klaus,Müller ^[7] ; Lucia Dora,Notarangelo ^[8] ; Davide,Montin ^[9] ; Joris M,van Montfrans ^[10] ; Maria Grazia,Roncarolo ^[3] ; Elisabetta,Traggiai ^[11] ; Jacques J M,van Dongen ^[4] ; Mirjam,van der Burg ^[4] ; Alessandro,Aiuti ^[12]

作者单位： San Raffaele Telethon Institute for Gene Therapy (TIGET), San Raffaele Scientific Institute, Milan, Italy. ^[1] Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy. ^[2] San Raffaele Telethon Institute for Gene Therapy (TIGET), San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy. ^[3] Department of Immunology, Erasmus MC, University Medical Center, Rotterdam, The Netherlands. ^[4] CUSSB, Vita-Salute San Raffaele University, Milan, Italy. ^[5] Division of Allergy, Immunology and Bone Marrow Transplantation, Department of Pediatrics, University of California San Francisco, San Francisco, Calif. ^[6] Pediatric Clinic, Juliane Marie Center, Copenhagen, Denmark. ^[7] Pediatric Onco-Hematology and BMT Unit, Children's Hospital, Spedali Civili, Brescia, Italy. ^[8] Department of Pediatrics, University of Turin, Turin, Italy. ^[9] Department of Pediatric Immunology and Infectious Diseases, University Medical Center Utrecht, Utrecht, The Netherlands. ^[10] Novartis Institute for Research in Biomedicine, Basel, Switzerland. ^[11] San Raffaele Telethon Institute for Gene Therapy (TIGET), San Raffaele Scientific Institute, Milan, Italy; Department of Systems Medicine, Tor Vergata University, Rome, Italy. Electronic address: aiuti.alessandro@hsr.it. ^[12]

关键词 B-cell development Gene therapy adenosine deaminase–deficient severe combined immunodeficiency antibodies