TRPML2 channel modulation by PI(3,5)P₂ and small-molecule agonists controls endosomal vesicle dynamics - 20/07/25

Abstract |
TRPML2 is an endolysosomal calcium-permeable channel gated by phosphatidylinositol 3,5-bisphosphate (PI(3,5)P₂). However, its subcellular localization and functional contribution to compartment-specific vesicle trafficking remain incompletely defined. In this study, we identify Rab4-positive recycling endosomes as a key site of TRPML2 activity and regulation. We further examined a PI(3,5)P₂-insensitive TRPML2 mutant (R310A), which exhibited reduced channel activity, accumulated perinuclear vesicles, and impaired Rab4 + endosomal motility. To restore channel function, we employed two selective small-molecule TRPML2 agonists, ML2-SA1 and ML2-SA2. Endolysosomal patch-clamp recordings confirmed that both compounds activate wild-type TRPML2 and effectively restore channel activity in the R310A mutant. Functional imaging further demonstrated that ML2-SA2 treatment rescues vesicle redistribution, reduces Golgi accumulation, and promotes peripheral vesicle dynamics. Notably, these effects were observed even in the absence of functional PI(3,5)P₂ gating, indicating that small-molecule agonists can bypass endogenous lipid regulation to restore TRPML2 activity. Our findings identify TRPML2 as a druggable ion channel whose activity is essential for maintaining Rab4-dependent vesicle trafficking. This study establishes a mechanistic link between phosphoinositide sensitivity, TRPML2 activation, and endosomal motility, and highlights a potential therapeutic strategy for correcting trafficking defects caused by impaired lipid signaling in immune cells or other pathophysiological contexts involving endosomal stress.
Le texte complet de cet article est disponible en PDF.Keywords : TRPML, Phosphoinositides (PIPs), Endosomal trafficking, Rab4, Host defense
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Vol 189
Article 118350- août 2025 Retour au numéroBienvenue sur EM-consulte, la référence des professionnels de santé.
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