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Successful Treatment With Ledipasvir/Sofosbuvir in an Infant With Severe Combined Immunodeficiency Caused by Adenosine Deaminase Deficiency With HCV Allowed Gene Therapy with Strimvelis.

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第一作者： Francesca,Tucci

第一单位： Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.

作者： Francesca,Tucci ^[1] ; Valeria,Calbi ^[1] ; Federica,Barzaghi ^[2] ; Maddalena,Migliavacca ^[1] ; Francesca,Ferrua ^[3] ; Maria Ester,Bernardo ^[4] ; Daniele,Canarutto ^[1] ; Giulia,Consiglieri ^[1] ; Salvatore,Recupero ^[1] ; Francesco,Calzatini ^[1] ; Michela,Gabaldo ^[5] ; Caterina,Lucano ^[5] ; Miriam,Casiraghi ^[4] ; Silvia,Darin ^[4] ; Francesca,Dionisio ^[5] ; Sarah,Marktel ^[6] ; Enza,Cestone ^[7] ; Renato,Finazzi ^[8] ; Giorgina,Mieli-Vergani ^[9] ; Enzo,Boeri ^[10] ; Jonathan,Appleby ^[11] ; Dalia,Abd Elaziz ^[12] ; Fabio,Ciceri ^[13] ; Alessandro,Aiuti ^[3] ; Maria Pia,Cicalese ^[4]

作者单位： Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy. ^[1] Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.;Tor Vergata University, Rome, Italy. ^[2] Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.;San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.;Vita Salute San Raffaele University, Milan, Italy. ^[3] Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.;San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy. ^[4] San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy. ^[5] Haematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy. ^[6] Dermatology Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy. ^[7] Unit of Hepatology, IRCCS San Raffaele Scientific Institute, Milan, Italy. ^[8] MowatLabs, Pediatric Liver, GI & Nutrition Centre, King's College Hospital, London, UK. ^[9] Department of Virology, IRCCS San Raffaele Scientific Institute, Milan, Italy. ^[10] GlaxoSmithKline, Stevenage, Hertfordshire, UK. ^[11] Department of Pediatrics, Faculty of Medicine, Cairo University, Cairo, Egypt. ^[12] San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.;Vita Salute San Raffaele University, Milan, Italy.;Haematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy. ^[13]