Lysosomal function modulation

Lysosomal function modulation refers to pharmacological or genetic interventions aimed at increasing or decreasing lysosomal activity, stability, or biogenesis. Lysosomes are membrane-bound organelles essential for degrading and recycling cellular waste, signaling, and energy metabolism. Dysregulation of lysosomal function is implicated in a wide range of diseases, including lysosomal storage disorders, neurodegenerative conditions, cancer, cardiovascular, inflammatory, and infectious diseases. Rather than being a single molecular entity, lysosomal function modulation encompasses targeting different lysosomal processes or regulators (such as TFEB, DCAF7, or lysosomal membrane stability) with small molecules, biologics, or enzyme replacement therapies. While preclinical and some clinical approaches have shown promise, broad modulation of lysosomal function carries significant safety risks due to the organelle’s central cellular role

Other names

Lysosome modulationLysosomal modulationLysosomal activity modulationModulation of lysosomes

Lysosomal membrane permeabilization (e.g., by chloroquine); Inhibition or activation of autophagy; Enhancement or inhibition of lysosomal enzyme function; Modulation of transcriptional regulators (e.g., TFEB stabilization/activation or degradation).