Rapamycin Autophagy Enhancement for Globotriaosylceramide Clearance

Rapamycin inhibits mTORC1 kinase activity leading to TFEB dephosphorylation and nuclear translocation, dramatically upregulating lysosomal biogenesis genes including LAMP1, LAMP2, and cathepsin D. Enhanced autophagy flux promotes clearance of accumulated Gb3 deposits while rapamycin's anti-inflammatory properties reduce chronic activation of microglia and astrocytes in Fabry neuropathy.