Matches in SemOpenAlex for { <https://semopenalex.org/work/W4310370102> ?p ?o ?g. }
- W4310370102 endingPage "1028" @default.
- W4310370102 startingPage "977" @default.
- W4310370102 abstract "Lysosomes act center-stage as executive cell machinery platforms for autophagy, quality-control, metabolic recycling and homeostatic regulation of nutrient and energy supply. Dynamically fusing with membrane structures, they also engulf exogenous molecules and particulates, including microbes. Inborn lysosomal diseases occur in ≈ 1 in 5000 live births but their protean clinico-pathological effects inform large fields of cell biology and medicine. Of > 80 lysosomal diseases, two-thirds injure the brain relentlessly. Targeted protein, substrate dissolution and brain-penetrant therapies already prevent, arrest - even reverse - some lysosomal diseases: late-stage translational development of pharmacological chaperones, stem-cell technology and gene therapies is advancing rapidly." @default.
- W4310370102 created "2022-12-09" @default.
- W4310370102 creator A5080995646 @default.
- W4310370102 date "2023-01-01" @default.
- W4310370102 modified "2023-10-16" @default.
- W4310370102 title "Lysosomal Diseases" @default.
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