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Items: 3

1.
Neurodevelopmental Changes in Excitatory Synaptic Structure and Function in the Cerebral Cortex of Sanfilippo Syndrome IIIA Mice
Chrissa A. Dwyer, Samantha L. Scudder, Ying Lin, Lara E. Dozier, Dustin Phan, Nicola J. Allen, Gentry N. Patrick, Jeffrey D. Esko
Sci Rep. 2017; 7: 46576. Published online 2017 Apr 18. doi: 10.1038/srep46576
PMCID:
PMC5394534
AbstractArticlePDF–1.6M
2.
Ataxia is the major neuropathological finding in arylsulfatase G-deficient mice: similarities and dissimilarities to Sanfilippo disease (mucopolysaccharidosis type III)
Björn Kowalewski, Peter Heimann, Theresa Ortkras, Renate Lüllmann-Rauch, Tomo Sawada, Steven U. Walkley, Thomas Dierks, Markus Damme
Hum Mol Genet. 2015 Apr 1; 24(7): 1856–1868. Published online 2014 Dec 1. doi: 10.1093/hmg/ddu603
PMCID:
PMC4355020
AbstractArticlePDF–1.9M
3.
Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy
Virginia Haurigot, Sara Marcó, Albert Ribera, Miguel Garcia, Albert Ruzo, Pilar Villacampa, Eduard Ayuso, Sònia Añor, Anna Andaluz, Mercedes Pineda, Gemma García-Fructuoso, Maria Molas, Luca Maggioni, Sergio Muñoz, Sandra Motas, Jesús Ruberte, Federico Mingozzi, Martí Pumarola, Fatima Bosch
J Clin Invest. 2013 Aug 1; 123(8): 3254–3271. Published online 2013 Jul 1. doi: 10.1172/JCI66778
PMCID:
PMC3726158
AbstractArticlePDF–12M

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