Search Results - "Winner, Leanne K"

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

“…Mucopolysaccharidosis type IIIA (MPS IIIA) is predominantly a disorder of the central nervous system, caused by a deficiency of sulfamidase (SGSH) with…”
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AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains by 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

“…Patients with mucopolysaccharidosis type IIIA (MPS IIIA) lack the lysosomal enzyme sulfamidase (SGSH), which is responsible for the degradation of heparan…”
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Aldosterone glucuronidation by human liver and kidney microsomes and recombinant UDP‐glucuronosyltransferases: Inhibition by NSAIDs by Knights, Kathleen M., Winner, Leanne K., Elliot, David J., Bowalgaha, Kushari, Miners, John O.

“…WHAT IS ALREADY KNOWN ABOUT THIS SUBJECT • Carboxylic acid NSAIDs are extensively glucuronidated as either the parent drug or hydroxylated metabolites and…”
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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 by Uchaipichat, Verawan, Winner, Leanne K., Mackenzie, Peter I., Elliot, David J., Williams, J Andrew, Miners, John O.

“…Aims Using the fluconazole–zidovudine (AZT) interaction as a model, to determine whether inhibition of UDP–glucuronosyltransferase (UGT) catalysed drug…”
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The ovine Type II Gaucher disease model recapitulates aspects of human brain disease by Winner, Leanne K, Beard, Helen, Karageorgos, Litsa, Smith, Nicholas J, Hopwood, John J, Hemsley, Kim M

“…Acute neuronopathic (type II) Gaucher disease (GD) is a devastating, untreatable neurological disorder resulting from mutations in the glucocerebrosidase gene…”
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Biomarkers for predicting disease course in Sanfilippo syndrome: An urgent unmet need in childhood‐onset dementia by Winner, Leanne K., Rogers, Mary‐Louise, Snel, Marten F., Hemsley, Kim M.

“…Sanfilippo syndrome (MPS III) is an autosomal recessive inherited disorder causing dementia in children, following an essentially normal early developmental…”
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Repetitive, non-invasive imaging of neurodegeneration, and prevention of it with gene replacement, in mice with Sanfilippo syndrome by 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

“…Hampering assessment of treatment outcomes in gene therapy and other clinical trials in patients with childhood dementia is the lack of an objective,…”
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Delivery of therapeutic protein for prevention of neurodegenerative changes: Comparison of different CSF-delivery methods by 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.

“…Injection of lysosomal enzyme into cisternal or ventricular cerebrospinal fluid (CSF) has been carried out in 11 lysosomal storage disorder models, with each…”
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Evaluation of Disease Lesions in the Developing Canine MPS IIIA Brain by Winner, Leanne K., Marshall, Neil R., Jolly, Robert D., Trim, Paul J., Duplock, Stephen K., Snel, Marten F., Hemsley, Kim M.

“…Mucopolysaccharidosis IIIA (MPS IIIA) is an inherited neurodegenerative disease of childhood that results in early death. Post-mortem studies have been carried…”
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Neonatal Bone Marrow Transplantation in MPS IIIA Mice by Lau, Adeline A, Shamsani, N. Jannah, Winner, Leanne K., Hassiotis, Sofia, King, Barbara M., Hopwood, John J., Hemsley, Kim M.

“…Patients with some neurological lysosomal storage disorders (LSD) exhibit improved clinical signs following bone marrow transplantation (BMT). The failure of…”
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