Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates.

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作者： Helena,Costa-Verdera ^[1] ; Fanny,Collaud ^[2] ; Christopher R,Riling ^[3] ; Pauline,Sellier ^[1] ; Jayme M L,Nordin ^[2] ; G Michael,Preston ^[4] ; Umut,Cagin ^[1] ; Julien,Fabregue ^[2] ; Simon,Barral ^[4] ; Maryse,Moya-Nilges ^[4] ; Jacomina,Krijnse-Locker ^[1] ; Laetitia,van Wittenberghe ^[2] ; Natalie,Daniele ^[1] ; Bernard,Gjata ^[2] ; Jeremie,Cosette ^[1] ; Catalina,Abad ^[2] ; Marcelo,Simon-Sola ^[5] ; Severine,Charles ^[5] ; Mathew,Li ^[1] ; Marco,Crosariol ^[1] ; Tom,Antrilli ^[1] ; William J,Quinn ^[1] ; David A,Gross ^[6] ; Olivier,Boyer ^[1] ; Xavier M,Anguela ^[2] ; Sean M,Armour ^[1] ; Pasqualina,Colella ^[2] ; Giuseppe,Ronzitti ^[4] ; Federico,Mingozzi ^[4]

作者单位： Genethon, 91000, Evry, France. ^[1] Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France. ^[2] Sorbonne University Paris and INSERM U974, 75013, Paris, France. ^[3] Spark Therapeutics, Philadelphia, PA, 19104, USA. ^[4] Pasteur Institute, 75015, Paris, France. ^[5] Université de Rouen Normandie-IRIB, 76183, Rouen, France. ^[6] Genethon, 91000, Evry, France. Federico.mingozzi@sparktx.com. ^[7] Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France. Federico.mingozzi@sparktx.com. ^[8] Sorbonne University Paris and INSERM U974, 75013, Paris, France. Federico.mingozzi@sparktx.com. ^[9] Spark Therapeutics, Philadelphia, PA, 19104, USA. Federico.mingozzi@sparktx.com. ^[10]