Safety and Efficacy of Liver-Directed Gene Therapy in Patients with Mucopolysaccharidosis Type VI
Nicola Brunetti-Pierri,Rita Ferla,Virginia M. Ginocchio,Alessandro Rossi,Simona Fecarotta,Roberta Romano,Giancarlo Parenti,Yilmaz Yildiz,Stefano Zancan, Valentina Pecorella, Mafalda Graziano,Margherita Dell'Anno,Marialuisa Alliegro,Francesca Santamaria,Raffaella Brunetti-Pierri,Francesca Simonelli,Vincenzo Nigro,Maria Vargas,Giuseppe Servillo,Francesco Borgia,Ernesto Soscia,Olivier Danos,Jean-Brice Marteau,Stefania Galimberti,Mariagrazia Valsecchi,Philippe Veron,Federico Mingozzi,Francesca Fallarino,Giancarlo La Marca,Alberto Auricchio MOLECULAR THERAPY（2021）
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