Fetal gene therapy for neurodegenerative disease of infants von Massaro, Giulia, Mattar, Citra N. Z., Wong, Andrew M. S., Sirka, Ernestas, Buckley, Suzanne M. K., Herbert, Bronwen R., Karlsson, Stefan, Perocheau, Dany P., Burke, Derek, Heales, Simon, Richard-Londt, Angela, Brandner, Sebastian, Huebecker, Mylene, Priestman, David A., Platt, Frances M., Mills, Kevin, Biswas, Arijit, Cooper, Jonathan D., Chan, Jerry K. Y., Cheng, Seng H., Waddington, Simon N., Rahim, Ahad A.

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Disruption of the autophagy-lysosome pathway is involved in neuropathology of the nclf mouse model of neuronal ceroid lipofuscinosis von Thelen, Melanie, Damme, Markus, Daμμe, Markus, Schweizer, Michaela, Hagel, Christian, Wong, Andrew M S, Cooper, Jonathan D, Braulke, Thomas, Galliciotti, Giovanna

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Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system von Rahim, Ahad A., Wong, Andrew M. S., Hoefer, Klemens, Buckley, Suzanne M. K., Mattar, Citra N., Cheng, Seng H., Chan, Jerry K. Y., Cooper, Jonathan D., Waddington, Simon N.

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Early glial activation, synaptic changes and axonal pathology in the thalamocortical system of Niemann–Pick type C1 mice von Pressey, Sarah N.R, Smith, David A, Wong, Andrew M.S, Platt, Frances M, Cooper, Jonathan D

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An anti-neuroinflammatory that targets dysregulated glia enhances the efficacy of CNS-directed gene therapy in murine infantile neuronal ceroid lipofuscinosis von Macauley, Shannon L, Wong, Andrew M S, Shyng, Charles, Augner, David P, Dearborn, Joshua T, Pearse, Yewande, Roberts, Marie S, Fowler, Stephen C, Cooper, Jonathan D, Watterson, D Martin, Sands, Mark S

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Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors von Karda, Rajvinder, Rahim, Ahad A., Wong, Andrew M. S., Suff, Natalie, Diaz, Juan Antinao, Perocheau, Dany P., Tijani, Maha, Ng, Joanne, Baruteau, Julien, Martin, Nuria Palomar, Hughes, Michael, Delhove, Juliette M. K. M., Counsell, John R., Cooper, Jonathan D., Henckaerts, Els, Mckay, Tristan R., Buckley, Suzanne M. K., Waddington, Simon N.

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Glial cells are functionally impaired in juvenile neuronal ceroid lipofuscinosis and detrimental to neurons von Parviainen, Lotta, Dihanich, Sybille, Anderson, Greg W, Wong, Andrew M, Brooks, Helen R, Abeti, Rosella, Rezaie, Payam, Lalli, Giovanna, Pope, Simon, Heales, Simon J, Mitchison, Hannah M, Williams, Brenda P, Cooper, Jonathan D

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Appalachian Nursing Homes: A Descriptive Analysis of Quality, Staffing, and Cost von Wong, Andrew M., Braun, Robert Tyler

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Systemic gene delivery following intravenous administration of AAV9 to fetal and neonatal mice and late‐gestation nonhuman primates von Mattar, Citra N., Wong, Andrew M. S., Hoefer, Klemens, Alonso‐Ferrero, Maria E., Buckley, Suzanne M. K., Howe, Steven J., Cooper, Jonathan D., Waddington, Simon N., Chan, Jerry K. Y., Rahim, Ahad A.

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DHHC5 Interacts with PDZ Domain 3 of Post-synaptic Density-95 (PSD-95) Protein and Plays a Role in Learning and Memory von Li, Yi, Hu, Jie, Höfer, Klemens, Wong, Andrew M.S., Cooper, Jonathan D., Birnbaum, Shari G., Hammer, Robert E., Hofmann, Sandra L.

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Intravenous high-dose enzyme replacement therapy with recombinant palmitoyl–protein thioesterase reduces visceral lysosomal storage and modestly prolongs survival in a preclinical... von Hu, Jie, Lu, Jui-Yun, Wong, Andrew M.S., Hynan, Linda S., Birnbaum, Shari G., Yilmaz, Denis S., Streit, Barbara M., Lenartowicz, Ewelina M., Thompson, Thomas C.M., Cooper, Jonathan D., Hofmann, Sandra L.

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Regional Brain Atrophy in Mouse Models of Neuronal Ceroid Lipofuscinosis: A New Rostrocaudal Perspective von Kühl, Thomas G., Dihanich, Sybille, Wong, Andrew M. S., Cooper, Jonathan D.

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Non-invasive molecular imaging of inflammatory macrophages in allograft rejection von O’Neill, Alexander S. G., Terry, Samantha Y. A., Brown, Kathryn, Meader, Lucy, Wong, Andrew M. S., Cooper, Jonathan D., Crocker, Paul R., Wong, Wilson, Mullen, Gregory E. D.

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Exacerbated neuronal ceroid lipofuscinosis phenotype in Cln1/5 double-knockout mice von Blom, Tea, Schmiedt, Mia-Lisa, Wong, Andrew M, Kyttälä, Aija, Soronen, Jarkko, Jauhiainen, Matti, Tyynelä, Jaana, Cooper, Jonathan D, Jalanko, Anu

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Behavioral deficits, early gliosis, dysmyelination and synaptic dysfunction in a mouse model of mucolipidosis IV von Grishchuk, Yulia, Sri, Sarmi, Rudinskiy, Nikita, Ma, Weiyuan, Stember, Katherine G, Cottle, Matthew W, Sapp, Ellen, Difiglia, Marian, Muzikansky, Alona, Betensky, Rebecca A, Wong, Andrew M S, Bacskai, Brian J, Hyman, Bradley T, Kelleher, 3rd, Raymond J, Cooper, Jonathan D, Slaugenhaupt, Susan A

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Proteomic mapping of differentially vulnerable pre-synaptic populations identifies regulators of neuronal stability in vivo von Llavero Hurtado, Maica, Fuller, Heidi R., Wong, Andrew M. S., Eaton, Samantha L., Gillingwater, Thomas H., Pennetta, Giuseppa, Cooper, Jonathan D., Wishart, Thomas M.

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Synergistic effects of central nervous system-directed gene therapy and bone marrow transplantation in the murine model of infantile neuronal ceroid lipofuscinosis von Macauley, Shannon L., Roberts, Marie S., Wong, Andrew M., McSloy, Francesca, Reddy, Adarsh S., Cooper, Jonathan D., Sands, Mark S.

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Nonclinical evaluation of CNS-administered TPP1 enzyme replacement in canine CLN2 neuronal ceroid lipofuscinosis von Vuillemenot, Brian R., Kennedy, Derek, Cooper, Jonathan D., Wong, Andrew M.S., Sri, Sarmi, Doeleman, Thom, Katz, Martin L., Coates, Joan R., Johnson, Gayle C., Reed, Randall P., Adams, Eric L., Butt, Mark T., Musson, Donald G., Henshaw, Joshua, Keve, Steve, Cahayag, Rhea, Tsuruda, Laurie S., O'Neill, Charles A.

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Treatment of HCV in renal transplant patients with peginterferon and ribavirin: long-term follow-up von Mak, Siu-ka, Sin, Ho-kwan, Lo, Kin-yee, Lo, Man-wai, Chan, Shuk-fan, Lo, Kwok-chi, Wong, Yuk-yi, Ho, Lo-yi, Wong, Ping-nam, Wong, Andrew K. M.

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Screening Algorithm for BK Virus-Associated Nephropathy Using Sequential Testing of Urinary Cytology: A Probabilistic Model Analysis von Ma, Maggie K.M., Leung, Anskar Y.H., Lo, Kin Yee, Lio, Weng In, Chan, Hoi Wong, Wong, Ivy, Hau, Anthony K.C., Tam, Chun Hay, Wong, Andrew K.M., Kuok, Un I, Chau, Ka Foon, Fung, Samuel K.S., Kwan, Tsz Hoi, Wong, Sunny S.H., Tang, Sydney C.W.

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