are the most widely used. However, despite these advancements, most lysosomal storage disorders still lack effective treatments as the existing ones are limited by poor efficacy and are typically disease specific.

Lysosomotropism

Lysosomotropism refers to the tendency of lipophilic weak bases to accumulate in acidic organelles like lysosomes. While neutral forms of these compounds cross membranes easily, their protonated (charged) forms become trapped inside lysosomes, leading to concentrations up to 1000 times higher than outside

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