Table 1 Lysosome as the therapeutic target in various diseases.
From: Lysosomal quality control of cell fate: a novel therapeutic target for human diseases
Diseases | Lysosome associated alterations | Consequenses | Intervention and therapy | |
|---|---|---|---|---|
Tumor | PDA | Upregulation of lysosomal and autophagic function by activation of TFEB | Catabolic hyperactivity and prone to invasion and progression | ATG5 or ATG7 knock out, hydroxychloroquine and its derivatives alone or in combination with standard chemotherapy drugs |
Breast cancer | Overexpression of cathepsin B | Downregulating the shRNA of cathepsin B | ||
Neurodegenerative diseases | GD | Abnormal transport of glucosinolates to lysosomes | Neurological dysfunction | Enzyme replacement therapy (injecting glucosinolidase or its structural analog) |
HSP | Damaged lysosomal biogenes caused by mutations in SPG11 and SPG15 genes, intracellular accumulation of gangliosides | Progressive spasm of the lower limbs caused by axial mutation of upper motor neurons | Alleviating the degradation pressure of lysosomes and improving lysosomal function | |
AD | Increased lysosomal pH and impaired lysosomal proteolytic function | AD-like pathologic change | ||
Inflammatory diseases | Acute hyperuricemia | Lysosomal membrane destroyed and lysosomal rupture by uric acid crystals | Renal inflammation | |
AP | Activation of cell death pathway by cathepsin B leaked into the cytoplasm | Pancreatic injury mainly in the form of cellular apoptosis and necrosis | ||
Sepsis | Upregulation of the key genes of lysosomes | Immunosuppression and multiple organ dysfunction | A variety of drugs used to reverse the immunosuppression of sepsis have been proved to be related to lysosomal and autophagic function | |
Cardiovascular diseases | AS | Decrease of LAL activity by excessive free cholesterol accumulated in lysosomes | Accumulation of cholesterol esters in lysosomes and cytoplasm in foam cells | Infusion of recombinant human LAL |
Danon disease | Deletion of LAMP2 gene | Lysosomal and autophagosome fusion disorders, massive autophagosome deposition in the cytoplasm of myocardium and skeletal muscle | Enhancing lysosomal function by TFEB overexpression | |
