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
Articles, Neurobiology of Disease
Intracranial Delivery of CLN2 Reduces Brain Pathology in a Mouse Model of Classical Late Infantile Neuronal Ceroid Lipofuscinosis
Marco A. Passini, James C. Dodge, Jie Bu, Wendy Yang, Qi Zhao, Dolan Sondhi, Neil R. Hackett, Stephen M. Kaminsky, Qinwen Mao, Lamya S. Shihabuddin, Seng H. Cheng, David E. Sleat, Gregory R. Stewart, Beverly L. Davidson, Peter Lobel and Ronald G. Crystal

Journal of Neuroscience 1 February 2006, 26 (5) 1334-1342; DOI: https://doi.org/10.1523/JNEUROSCI.2676-05.2006
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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lysosomal storage disease