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Plos One|January 19, 2019
BMN 250, a fusion of lysosomal alpha-N-acetylglucosaminidase with IGF2, exhibits different patterns of cellular uptake into critical cell types of Sanfilippo syndrome B disease pathogenesisGouri Yogalingam, Amanda R Luu, Heather Prill, et al.
Molecular Genetics and Metabolism Reports|November 14, 2019
Characterization of glycan substrates accumulating in GM1 GangliosidosisRoger Lawrence, Jeremy L Van Vleet, Linley Mangini, et al.
Plos One|December 15, 2010
Genistein improves neuropathology and corrects behaviour in a mouse model of neurodegenerative metabolic diseaseMarcelina Malinowska, Fiona L Wilkinson, Kia J Langford-Smith, et al.
Stem Cells (Dayton, Ohio)|April 28, 2007
Essential alterations of heparan sulfate during the differentiation of embryonic stem cells to Sox1-enhanced green fluorescent protein-expressing neural progenitor cellsClaire E Johnson, Brett E Crawford, Marios Stavridis, et al.
Nucleic Acid Therapeutics|February 7, 2025
Targeting a Novel Site in Exon 51 with Antisense Oligonucleotides Induces Enhanced Exon Skipping in a Mouse Model of Duchenne Muscular DystrophyTodd Oppeneer, Yulan Qi, Joshua Henshaw, et al.
Scientific Reports|November 24, 2020
Central nervous system pathology in preclinical MPS IIIB dogs reveals progressive changes in clinically relevant brain regionsMartin T Egeland, Marta M Tarczyluk-Wells, Melissa M Asmar, et al.
Scientific Reports|July 15, 2021
Substrate reduction therapy for Krabbe disease and metachromatic leukodystrophy using a novel ceramide galactosyltransferase inhibitorMichael C Babcock, Christina R Mikulka, Bing Wang, et al.
Proceedings of the National Academy of Sciences of the United States of America|September 19, 2019
Genetic ablation of acid ceramidase in Krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic targetYedda Li, Yue Xu, Bruno A Benitez, 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.
The Journal of Biological Chemistry|September 5, 2019
Intracerebroventricular enzyme replacement therapy with β-galactosidase reverses brain pathologies due to GM1 gangliosidosis in miceJoseph C Chen, Amanda R Luu, Nathan Wise, et al.
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