Early glial activation, synaptic changes and axonal pathology in the thalamocortical system of Niemann–Pick type C1 mice

Autor Pressey, Sarah N.R., Smith, David A., Wong, Andrew M.S., Platt, Frances M., Cooper, Jonathan D.
Vydáno 2012

Correction: Disruption of the Autophagy-Lysosome Pathway Is Involved in Neuropathology of the nclf Mouse Model of Neuronal Ceroid Lipofuscinosis

Autor Thelen, Melanie, Damme, Markus, Schweizer, Michaela, Hagel, Christian, Wong, Andrew M.S., Cooper, Jonathan D., Braulke, Thomas, Galliciotti, Giovanna
Vydáno 2012

Disruption of the Autophagy-Lysosome Pathway Is Involved in Neuropathology of the nclf Mouse Model of Neuronal Ceroid Lipofuscinosis

Autor Thelen, Melanie, Daμμe, Markus, Schweizer, Michaela, Hagel, Christian, Wong, Andrew M.S., Cooper, Jonathan D., Braulke, Thomas, Galliciotti, Giovanna
Vydáno 2012

Immunosuppression alters disease severity in juvenile Batten disease mice

Autor Seehafer, Sabrina S., Ramirez-Montealegre, Denia, Wong, Andrew MS, Chan, Chun-Hung, Castaneda, Julian, Horak, Michael, Ahmadi, Sarah M, Lim, Ming J, Cooper, Jonathan D, Pearce, David A
Vydáno 2011

An Anti-Neuroinflammatory That Targets Dysregulated Glia Enhances the Efficacy of CNS-Directed Gene Therapy in Murine Infantile Neuronal Ceroid Lipofuscinosis

Autor 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.
Vydáno 2014

Partial Correction of the CNS Lysosomal Storage Defect in a Mouse Model of Juvenile Neuronal Ceroid Lipofuscinosis by Neonatal CNS Administration of an Adeno-Associated Virus Serot...

Autor Sondhi, Dolan, Scott, Emma C., Chen, Alvin, Hackett, Neil R., Wong, Andrew M.S., Kubiak, Agnieszka, Nelvagal, Hemanth R., Pearse, Yewande, Cotman, Susan L., Cooper, Jonathan D., Crystal, Ronald G.
Vydáno 2014

Intravenous high-dose enzyme replacement therapy with recombinant palmitoyl-protein thioesterase reduces visceral lysosomal storage and modestly prolongs survival in a preclinical...

Autor 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.
Vydáno 2012

A novel, long-lived, and highly engraftable immunodeficient mouse model of mucopolysaccharidosis type I

Autor Mendez, Daniel C, Stover, Alexander E, Rangel, Anthony D, Brick, David J, Nethercott, Hubert E, Torres, Marissa A, Khalid, Omar, Wong, Andrew MS, Cooper, Jonathan D, Jester, James V, Monuki, Edwin S, McGuire, Cian, Le, Steven Q, Kan, Shih-hsin, Dickson, Patricia I, Schwartz, Philip H
Vydáno v Mol Ther Methods Clin Dev (2015)

Fetal gene therapy for neurodegenerative disease of infants

Autor 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.
Vydáno v Nat Med (2018)