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AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains.

Autor: Hocquemiller M; Lysogene, 18-20 rue Jacques Dulud, 92200 Neuilly-sur-Seine, France., Hemsley KM; Childhood Dementia Research Group, Hopwood Centre for Neurobiology, Lifelong Health Theme, South Australian Health and Medical Research Institute, Adelaide, SA 5000, Australia., Douglass ML; Childhood Dementia Research Group, Hopwood Centre for Neurobiology, Lifelong Health Theme, South Australian Health and Medical Research Institute, Adelaide, SA 5000, Australia., Tamang SJ; Childhood Dementia Research Group, Hopwood Centre for Neurobiology, Lifelong Health Theme, South Australian Health and Medical Research Institute, Adelaide, SA 5000, Australia., Neumann D; Childhood Dementia Research Group, Hopwood Centre for Neurobiology, Lifelong Health Theme, South Australian Health and Medical Research Institute, Adelaide, SA 5000, Australia., King BM; Childhood Dementia Research Group, Hopwood Centre for Neurobiology, Lifelong Health Theme, South Australian Health and Medical Research Institute, Adelaide, SA 5000, Australia., Beard H; Childhood Dementia Research Group, Hopwood Centre for Neurobiology, Lifelong Health Theme, South Australian Health and Medical Research Institute, Adelaide, SA 5000, Australia., Trim PJ; Mass Spectrometry Core Facility, SAHMRI, Adelaide, SA 5000, Australia., Winner LK; Childhood Dementia Research Group, Hopwood Centre for Neurobiology, Lifelong Health Theme, South Australian Health and Medical Research Institute, Adelaide, SA 5000, Australia., Lau AA; Childhood Dementia Research Group, Hopwood Centre for Neurobiology, Lifelong Health Theme, South Australian Health and Medical Research Institute, Adelaide, SA 5000, Australia., Snel MF; Mass Spectrometry Core Facility, SAHMRI, Adelaide, SA 5000, Australia., Gomila C; Laboratoire de Biochimie Métabolique, CHU Amiens Picardie, 80054 Amiens, France., Ausseil J; Unité INSERM U1043, Centre de Physiopathologie Toulouse Purpan (CPTP), Université Paul Sabatier, 31024 Toulouse, France., Mei X; Lysogene, 18-20 rue Jacques Dulud, 92200 Neuilly-sur-Seine, France., Giersch L; Lysogene, 18-20 rue Jacques Dulud, 92200 Neuilly-sur-Seine, France., Plavsic M; Lysogene, 18-20 rue Jacques Dulud, 92200 Neuilly-sur-Seine, France., Laufer R; Lysogene, 18-20 rue Jacques Dulud, 92200 Neuilly-sur-Seine, France.

Publikováno v: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2019 Dec 10; Vol. 17, pp. 174-187. Date of Electronic Publication: 2019 Dec 10 (Print Publication: 2020).