Endo‐lysosomal and autophagic dysfunction: a driving factor in Alzheimer's disease? von Whyte, Lauren S., Lau, Adeline A., Hemsley, Kim M., Hopwood, John J., Sargeant, Timothy 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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Compromised transcription-mRNA export factor THOC2 causes R-loop accumulation, DNA damage and adverse neurodevelopment von Bhattacharjee, Rudrarup, Jolly, Lachlan A., Corbett, Mark A., Wee, Ing Chee, Rao, Sushma R., Gardner, Alison E., Ritchie, Tarin, van Hugte, Eline J. H., Ciptasari, Ummi, Piltz, Sandra, Noll, Jacqueline E., Nazri, Nazzmer, van Eyk, Clare L., White, Melissa, Fornarino, Dani, Poulton, Cathryn, Baynam, Gareth, Collins-Praino, Lyndsey E., Snel, Marten F., Nadif Kasri, Nael, Hemsley, Kim M., Thomas, Paul Q., Kumar, Raman, Gecz, Jozef

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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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Determination of the role of injection site on the efficacy of intra-CSF enzyme replacement therapy in MPS IIIA mice von Beard, Helen, Luck, Amanda J., Hassiotis, Sofia, King, Barbara, Trim, Paul J., Snel, Marten F., Hopwood, John J., Hemsley, Kim M.

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Is the eye a window to the brain in Sanfilippo syndrome? von Beard, Helen, Chidlow, Glyn, Neumann, Daniel, Nazri, Nazzmer, Douglass, Meghan, Trim, Paul J, Snel, Marten F, Casson, Robert J, Hemsley, Kim M

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Reduction in open field activity in the absence of memory deficits in the AppNL−G−F knock-in mouse model of Alzheimer’s disease von Whyte, Lauren S., Hemsley, Kim M., Lau, Adeline A., Hassiotis, Sofia, Saito, Takashi, Saido, Takaomi C., Hopwood, John J., Sargeant, Timothy J.

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Enzyme replacement reduces neuropathology in MPS IIIA dogs von Crawley, Allison C, Marshall, Neil, Beard, Helen, Hassiotis, Sofia, Walsh, Vicki, King, Barbara, Hucker, Nicola, Fuller, Maria, Jolly, Robert D, Hopwood, John J, Hemsley, Kim M

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Open field locomotor activity and anxiety-related behaviors in mucopolysaccharidosis type IIIA mice von Lau, Adeline A., Crawley, Allison C., Hopwood, John J., Hemsley, Kim M.

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Examination of intravenous and intra-CSF protein delivery for treatment of neurological disease von Hemsley, Kim M., Luck, Amanda J., Crawley, Allison C., Hassiotis, Sofia, Beard, Helen, King, Barbara, Rozek, Tomas, Rozaklis, Tina, Fuller, Maria, Hopwood, John J.

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Lysosomal gene Hexb displays haploinsufficiency in a knock-in mouse model of Alzheimer’s disease von Whyte, Lauren S., Fourrier, Célia, Hassiotis, Sofia, Lau, Adeline A., Trim, Paul J., Hein, Leanne K., Hattersley, Kathryn J., Bensalem, Julien, Hopwood, John J., Hemsley, Kim M., Sargeant, Timothy J.

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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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Axonal dystrophy in the brain of mice with Sanfilippo syndrome von Beard, Helen, Hassiotis, Sofia, Gai, Wei-Ping, Parkinson-Lawrence, Emma, Hopwood, John J., 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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Filipin complex‐reactive brain lesions: A cautionary tale von Lau, Adeline A., Trim, Paul J., King, Barbara M., Hassiotis, Sofia, Hung, Ya Hui, Bush, Ashley I., Snel, Marten F., Hemsley, Kim M.

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Lysosomal Dysregulation in the Murine AppNL-G-F/NL-G-F Model of Alzheimer’s Disease von Whyte, Lauren S., Hassiotis, Sofia, Hattersley, Kathryn J., Hemsley, Kim M., Hopwood, John J., Lau, Adeline A., Sargeant, Timothy J.

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Adeno-associated viral gene therapy for mucopolysaccharidoses exhibiting neurodegeneration von Lau, Adeline A., Hemsley, Kim M.

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MPS-IIIA mice acquire autistic behaviours with age von Lau, Adeline A., Tamang, Sarah J., Hemsley, Kim M.

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Drosophila melanogaster models of MPS IIIC (Hgsnat‐deficiency) highlight the role of glia in disease presentation von Hewson, Laura, Choo, Amanda, Webber, Dani L., Trim, Paul J., Snel, Marten F., Fedele, Anthony O., Hopwood, John J., Hemsley, Kim M., O'Keefe, Louise V.

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