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Overall, transporter-mediated strategies leverage endogenous carrier systems (such as GLUT1 for glucose, LAT1 for large neutrals) to shuttle therapeutics across the BBB, offering distinct advantages: (1) High specificity through natural substrate-receptor interactions, minimizing off-target effects
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Familial forms of neurodegenerative lysosomal storage disorders (LSD) like Gaucher, Krabbe, Niemann-Pick type 1 and Fabry are caused by mutations in genes encoding enzymes that function in the sphingolipid pathway [179][180][181]
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