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Bio-Protocol|August 23, 2016
Combined <i>in situ</i> Hybridization/Immunohistochemistry (ISH/IH) on Free-floating Vibratome Tissue SectionsManuel E LopezDisease Models & Mechanisms|August 3, 2013
Genetic dissection of a cell-autonomous neurodegenerative disorder: lessons learned from mouse models of Niemann-Pick disease type CManuel E Lopez, Matthew P ScottJournal of Neuroinflammation|September 19, 2012
Complement is dispensable for neurodegeneration in Niemann-Pick disease type CManuel E Lopez, Andres D Klein, Matthew P ScottThe Journal of Neuroscience : the Official Journal of the Society for Neuroscience|March 25, 2011
Anatomically defined neuron-based rescue of neurodegenerative Niemann-Pick type C disorderManuel E Lopez, Andres D Klein, Ubah J Dimbil, et al.Human Molecular Genetics|April 12, 2012
Neuronal and epithelial cell rescue resolves chronic systemic inflammation in the lipid storage disorder Niemann-Pick CManuel E Lopez, Andrés D Klein, Jennifer Hong, et al.JIMD Reports|July 8, 2024
Galactokinase 1 is the source of elevated galactose-1-phosphate and cerebrosides are modestly reduced in a mouse model of classic galactosemiaLinley Mangini, Roger Lawrence, Manuel E Lopez, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|February 8, 2024
Prenatal delivery of a therapeutic antisense oligonucleotide achieves broad biodistribution in the brain and ameliorates Angelman syndrome phenotype in miceMaria T Clarke, Laura Remesal, Lea Lentz, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|September 24, 2025
AAV delivery of full-length SYNGAP1 rescues epileptic and behavioral phenotypes in a mouse model of SYNGAP1-related disordersMeagan A Quinlan, Rong Guo, Andrew G Clark, et al.Plos One|January 3, 2025
Intracerebroventricular administration of a modified hexosaminidase ameliorates late-stage neurodegeneration in a GM2 mouse modelManuel E Lopez, Daniel Wendt, Roger Lawrence, et al.Pageof 1