The ovine Type II Gaucher disease model recapitulates aspects of human brain disease von Winner, Leanne K., Beard, Helen, Karageorgos, Litsa, Smith, Nicholas J., Hopwood, John J., Hemsley, Kim M.

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Biomarkers for predicting disease course in Sanfilippo syndrome: An urgent unmet need in childhood‐onset dementia von Winner, Leanne K., Rogers, Mary‐Louise, Snel, Marten F., Hemsley, Kim M.

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Repetitive, non-invasive imaging of neurodegeneration, and prevention of it with gene replacement, in mice with Sanfilippo syndrome von Hemsley, Kim M, Beard, Helen, Chidlow, Glyn, Mammone, Teresa, Winner, Leanne K, Neumann, Daniel, King, Barbara, Snel, Marten F, Trim, Paul J, Casson, Robert J

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A Preclinical Study Evaluating AAVrh10-Based Gene Therapy for Sanfilippo Syndrome von Winner, Leanne K, Beard, Helen, Hassiotis, Sofia, Lau, Adeline A, Luck, Amanda J, Hopwood, John J, Hemsley, Kim M

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AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains von Hocquemiller, Michaël, Hemsley, Kim M., Douglass, Meghan L., Tamang, Sarah J., Neumann, Daniel, King, Barbara M., Beard, Helen, Trim, Paul J., Winner, Leanne K., Lau, Adeline A., Snel, Marten F., Gomila, Cathy, Ausseil, Jérôme, Mei, Xin, Giersch, Laura, Plavsic, Mark, Laufer, Ralph

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Quantitative prediction of in vivo inhibitory interactions involving glucuronidated drugs from in vitro data: the effect of fluconazole on zidovudine glucuronidation von Uchaipichat, Verawan, Winner, Leanne K., Mackenzie, Peter I., Elliot, David J., Williams, J Andrew, Miners, John O.

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Aldosterone glucuronidation by human liver and kidney microsomes and recombinant UDP‐glucuronosyltransferases: Inhibition by NSAIDs von Knights, Kathleen M., Winner, Leanne K., Elliot, David J., Bowalgaha, Kushari, Miners, John O.

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

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Evaluation of Disease Lesions in the Developing Canine MPS IIIA Brain von Winner, Leanne K., Marshall, Neil R., Jolly, Robert D., Trim, Paul J., Duplock, Stephen K., Snel, Marten F., Hemsley, Kim M.

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Superior outcomes in neuroretina following IV versus intra-CSF AAV9 gene replacement in mice with MPS IIIA von Hemsley, Kim M., Winner, Leanne, Shoubridge, Andrew, Parkinson-Lawrence, Emma, Lau, Adeline, Lance, Tabitha-Rose, King, Barbara, Scott, William, Snel, Marten, Trim, Paul, Beard, Helen

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Evaluation of neuroretina following i.v. or intra‐CSF AAV9 gene replacement in mice with MPS IIIA, a childhood dementia von Beard, Helen, Winner, Leanne, Shoubridge, Andrew, Parkinson‐Lawrence, Emma, Lau, Adeline A., Mubarokah, Siti N., Lance, Tabitha‐Rose, King, Barbara, Scott, William, Snel, Marten F., Trim, Paul J., Hemsley, Kim M.

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Neonatal Bone Marrow Transplantation in MPS IIIA Mice von Lau, Adeline A, Shamsani, N. Jannah, Winner, Leanne K., Hassiotis, Sofia, King, Barbara M., Hopwood, John J., Hemsley, Kim M.

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