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The EMBO Journal|December 31, 2002
Cathepsin A regulates chaperone-mediated autophagy through cleavage of the lysosomal receptorAna Maria Cuervo, Linda Mann, Erik J Bonten, et al.
Journal of Cellular Physiology|January 1, 2009
Chaperone-mediated autophagy is defective in mucolipidosis type IVBhuvarahamurthy Venugopal, Nicholas T Mesires, John C Kennedy, et al.
Traffic (Copenhagen, Denmark)|September 30, 2005
Unifying nomenclature for the isoforms of the lysosomal membrane protein LAMP-2Eeva-Liisa Eskelinen, Ana Maria Cuervo, Matthew R G Taylor, et al.
Cells|December 17, 2024
Macrophage Inhibitor Clodronate Enhances Liver Transduction of Lentiviral but Not Adeno-Associated Viral Vectors or mRNA Lipid Nanoparticles in Neonatal and Juvenile MiceLoukia Touramanidou, Sonam Gurung, Claudiu A Cozmescu, et al.
F1000Research|April 15, 2024
Ex vivo precision-cut liver slices model disease phenotype and monitor therapeutic response for liver monogenic diseasesDany Perocheau, Sonam Gurung, Loukia Touramanidou, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|January 12, 2025
Characterization of a novel conditional knockout mouse model to assess efficacy of mRNA therapy in the context of severe OTC deficiencyJenny Zhou, Shi Liang, Ling Yin, et al.
Molecular Therapy. Methods & Clinical Development|February 14, 2024
Whole-body galactose oxidation as a robust functional assay to assess the efficacy of gene-based therapies in a mouse model of GalactosemiaBijina Balakrishnan, Xinhua Yan, Marshall D McCue, et al.
Amino Acids|March 22, 2023
Amnio acid substitution at position 298 of human glucose-6 phosphatase-α significantly impacts its stability in mammalian cellsJingsong Cao, Arianna Markel, Erin Hanahoe, et al.
Molecular Genetics and Metabolism|June 18, 2010
Inhibition of glycogen biosynthesis via mTORC1 suppression as an adjunct therapy for Pompe diseaseKaren M Ashe, Kristin M Taylor, Qiuming Chu, et al.
American Journal of Human Genetics|March 19, 2019
Systemic mRNA Therapy for the Treatment of Fabry Disease: Preclinical Studies in Wild-Type Mice, Fabry Mouse Model, and Wild-Type Non-human PrimatesXuling Zhu, Ling Yin, Matt Theisen, et al.
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