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Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches.

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第一作者： Alessandro,Fraldi

第一单位： Telethon Institute of Genetic and Medicine (TIGEM), Via Campi Flegrei, 34, Pozzuoli, Naples, Italy. fraldi@tigem.it.;Department of Medical and Translational Science, Federico II University, Via Pansini 5, Naples, 80131, Italy. fraldi@tigem.it.

作者： Alessandro,Fraldi ^[1] ; Marta,Serafini ^[2] ; Nicolina Cristina,Sorrentino ^[3] ; Bernhard,Gentner ^[4] ; Alessandro,Aiuti ^[5] ; Maria Ester,Bernardo ^[6]

作者单位： Telethon Institute of Genetic and Medicine (TIGEM), Via Campi Flegrei, 34, Pozzuoli, Naples, Italy. fraldi@tigem.it.;Department of Medical and Translational Science, Federico II University, Via Pansini 5, Naples, 80131, Italy. fraldi@tigem.it. ^[1] Department of Pediatrics, Dulbecco Telethon Institute, Centro Ricerca M. Tettamanti, University of Milano-Bicocca, Monza, Italy. ^[2] Telethon Institute of Genetic and Medicine (TIGEM), Via Campi Flegrei, 34, Pozzuoli, Naples, Italy. ^[3] San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Via Olgettina, 60, 20123, Milan, Italy. ^[4] San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Via Olgettina, 60, 20123, Milan, Italy. aiuti.alessandro@hsr.it.;Unit of Pediatric Immunohematology and Stem Cell Program, IRCCS San Raffaele Scientific Institute, Via Olgettina, 60, 20123, Milan, Italy. aiuti.alessandro@hsr.it.;Vita Salute San Raffaele University, Milan, Italy. aiuti.alessandro@hsr.it. ^[5] San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Via Olgettina, 60, 20123, Milan, Italy.;Unit of Pediatric Immunohematology and Stem Cell Program, IRCCS San Raffaele Scientific Institute, Via Olgettina, 60, 20123, Milan, Italy. ^[6]

医学主题词人类(Humans);黏多糖累积病(Mucopolysaccharidoses)

DOI 10.1186/s13052-018-0565-y

PMID 30442177

发布时间 2023-11-04

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