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lysosomal storage disease

  • Articles, Neurobiology of Disease
    Self-Complementary AAV9 Gene Delivery Partially Corrects Pathology Associated with Juvenile Neuronal Ceroid Lipofuscinosis (CLN3)
    Megan E. Bosch, Amy Aldrich, Rachel Fallet, Jessica Odvody, Maria Burkovetskaya, Kaitlyn Schuberth, Julie A. Fitzgerald, Kevin D. Foust and Tammy Kielian
    Journal of Neuroscience 14 September 2016, 36 (37) 9669-9682; DOI: https://doi.org/10.1523/JNEUROSCI.1635-16.2016
  • Articles, Neurobiology of Disease
    Mechanism-Based Combination Treatment Dramatically Increases Therapeutic Efficacy in Murine Globoid Cell Leukodystrophy
    Jacqueline A. Hawkins-Salsbury, Lauren Shea, Xuntian Jiang, Daniel A. Hunter, A. Miguel Guzman, Adarsh S. Reddy, Elizabeth Y. Qin, Yedda Li, Steven J. Gray, Daniel S. Ory and Mark S. Sands
    Journal of Neuroscience 22 April 2015, 35 (16) 6495-6505; DOI: https://doi.org/10.1523/JNEUROSCI.4199-14.2015
  • Brief Communications
    An Anti-Neuroinflammatory That Targets Dysregulated Glia Enhances the Efficacy of CNS-Directed Gene Therapy in Murine Infantile Neuronal Ceroid Lipofuscinosis
    Shannon L. Macauley, Andrew M.S. Wong, Charles Shyng, David P. Augner, Joshua T. Dearborn, Yewande Pearse, Marie S. Roberts, Stephen C. Fowler, Jonathan D. Cooper, D. Martin Watterson and Mark S. Sands
    Journal of Neuroscience 24 September 2014, 34 (39) 13077-13082; DOI: https://doi.org/10.1523/JNEUROSCI.2518-14.2014
  • Featured ArticleArticles, Neurobiology of Disease
    Comparison of Five Peptide Vectors for Improved Brain Delivery of the Lysosomal Enzyme Arylsulfatase A
    Annika Böckenhoff, Sandra Cramer, Philipp Wölte, Simeon Knieling, Claudia Wohlenberg, Volkmar Gieselmann, Hans-Joachim Galla and Ulrich Matzner
    Journal of Neuroscience 26 February 2014, 34 (9) 3122-3129; DOI: https://doi.org/10.1523/JNEUROSCI.4785-13.2014
  • ARTICLE, Cellular/Molecular
    Distribution of a Lysosomal Enzyme in the Adult Brain by Axonal Transport and by Cells of the Rostral Migratory Stream
    Marco A. Passini, Edward B. Lee, Gregory G. Heuer and John H. Wolfe
    Journal of Neuroscience 1 August 2002, 22 (15) 6437-6446; DOI: https://doi.org/10.1523/JNEUROSCI.22-15-06437.2002
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