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Lysosome function and autophagy

Molecular classification
Organelle (lysosome), Biological process (autophagy, macroautophagy, microautophagy, chaperone-mediated autophagy), Enzyme (lysosomal hydrolases, e.g., cathepsins), Degradation pathway (lysosome-dependent degradation)
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Overview

Lysosome function and autophagy comprise key cellular processes by which cells degrade and recycle cytoplasmic constituents. Lysosomes are acidic organelles containing diverse hydrolases essential for the breakdown of proteins, organelles, and pathogens. Autophagy refers to the delivery of cytosolic material to lysosomes for degradation; this is subdivided into macroautophagy (autophagosome-dependent), microautophagy (direct invagination), and chaperone-mediated autophagy (substrate-selective). These pathways are fundamental for maintaining cellular homeostasis, adapting to stress, and defending against infections. Defects in autophagy or lysosomal function contribute to a range of human diseases, including cancer, neurodegeneration, infection, and myopathies. While many drugs can modulate these processes, and many lysosomal and autophagy proteins are valid therapeutic targets, "lysosome function / autophagy" itself is not a singular, druggable molecule or receptor but rather describes a broad, systemic biological axis.

Other names
autophagocytosisself-eatingautophagic process
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Mechanism of action

Inhibition of autophagosome–lysosome fusion; Inhibition or enhancement of lysosomal acidification; Induction or suppression of autophagy signaling pathways (e.g., mTOR, AMPK); Modulation of lysosomal hydrolase activity

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Biological functions

Cellular degradation and recyclingProtein and organelle quality controlCellular adaptation to stress/starvationImmune defense (clearance of pathogens)Regulation of cell death and survival
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Disease associations

CancerNeurodegenerative diseaseInfectionMyopathiesOther (inflammatory conditions, metabolic disease)
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Safety considerations

Excessive autophagy may promote cell deathChronic lysosome inhibition may be toxicModulation of autophagy can impair immune response or worsen some disease phenotypesOff-target effects due to broad involvement of autophagy in multiple physiological systems
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Interacting drugs

Chloroquine/hydroxychloroquine (inhibit lysosomal acidification, block autophagy flux)

3 more in the full profile.

07

Biomarkers

LC3 conversion (from LC3-I to LC3-II; marker of autophagosome formation)p62/SQSTM1 levels (marker of cargo degradation through autophagy)Autophagic flux assays

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