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Zoledronic acid ameliorates multiple organ pathologies in a murine model of mucolipidosis II by partially restoring mannose-6-phosphate-mediated lysosomal hydrolase activities
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DOI：10.1016/j.ymgme.2026.110191.png)
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Abstract
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• A Gnptab p.R364X knock-in mouse was developed, which recapitulates key features of human MLII. • LysoIP-based proteomics reveals global lysosomal remodeling in MLII murine fibroblasts, including depletion or decrease of M6P-dependent proteins and increase of M6P-independent hydrolases and integral membrane proteins in MLII lysosomes. • Beside skeletal system, Zoledronic acid ameliorates multiple organ pathologies of MLII mice, possibly through partially restoring intracellular M6P-dependent proteins. • Zoledronic acid increases activities of lysosomal luminal M6P-dependent lysosomal proteins and decreases intracellular glycosaminoglycan storage, providing a mechanistic basis for its specific therapeutic effects in MLII.
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