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Molecular Genetics and Metabolism|September 17, 2025
Defining lung pathogenesis in a murine model of mucopolysaccharidosis Type I by proteomic analysisYuen T Ngai, Clifford Young, Emma J Parkinson-Lawrence, et al.CNS Neuroscience & Therapeutics|August 10, 2024
Evaluation of neuroretina following i.v. or intra-CSF AAV9 gene replacement in mice with MPS IIIA, a childhood dementiaHelen Beard, Leanne Winner, Andrew Shoubridge, et al.Experimental Neurology|September 24, 2014
Delivery of therapeutic protein for prevention of neurodegenerative changes: comparison of different CSF-delivery methodsNeil R Marshall, Sofia Hassiotis, Barbara King, et al.Journal of Inherited Metabolic Disease|April 29, 2017
A novel conditional Sgsh knockout mouse model recapitulates phenotypic and neuropathic deficits of Sanfilippo syndromeAdeline A Lau, Barbara M King, Carly L Thorsen, et al.Molecular Therapy. Methods & Clinical Development|January 8, 2020
AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal BrainsMichaël Hocquemiller, Kim M Hemsley, Meghan L Douglass, et al.Nature Communications|April 7, 2026
Modelling synaptic dysfunction in childhood dementia using human iPSC-derived cortical networksParis Mazzachi, Ella McDonald, Zarina Greenberg, et al.Pageof 3