The ovine Type II Gaucher disease model recapitulates aspects of human brain disease.

Autor: Winner, Leanne K., Beard, Helen, Karageorgos, Litsa, Smith, Nicholas J., Hopwood, John J., Hemsley, Kim M.

Publikováno v: In BBA - Molecular Basis of Disease April 2023 1869(4)

Delivery of therapeutic protein for prevention of neurodegenerative changes: Comparison of different CSF-delivery methods

Autor: Marshall, Neil R., Hassiotis, Sofia, King, Barbara, Rozaklis, Tina, Trim, Paul J., Duplock, Stephen K., Winner, Leanne K., Beard, Helen, Snel, Marten F., Jolly, Robert D., Hopwood, John J., Hemsley, Kim M.

Publikováno v: In Experimental Neurology January 2015 263:79-90

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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).