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Target intelligence / Profile preview
Transient Receptor Potential Mucolipin 1 (TRPML1)
- Target
- TRPML1
- Molecular classification
- Ion channel, TRP ion channel family, Mucolipin subfamily (TRPML)
01
Overview
TRPML1 is a non-selective cation channel primarily localized in endosomes and lysosomes. It plays a crucial role in lysosomal calcium release, iron transport, vesicular trafficking, autophagy, and pH homeostasis. Loss-of-function mutations cause mucolipidosis type IV, while altered expression is implicated in cancer and neurodegenerative diseases. It functions as a sensor for reactive oxygen species and can activate either protective or apoptotic pathways depending on cellular context. MLSA1 is a known agonist.
- Other names
- MCOLN1
02
Mechanism of action
Agonist (MLSA1) activates TRPML1 channels leading to increased LC3-II expression and reduced ROS/apoptosis.
03
Biological functions
Ion transportLysosome homeostasisVesicular traffickingExocytosisAutophagy regulationROS sensingmTOR signaling regulationpH homeostasis
04
Disease associations
Mucolipidosis type IVCancerNeurodegenerative diseaseIron-deficiency anemia
05
Safety considerations
Modulating TRPML1 could have complex effects depending on the cellular context and level of oxidative stress, potentially leading to either cell survival or cell death.Given its role in lysosomal function, dysregulation could lead to lysosomal storage disorders or other cellular dysfunction.
06
Interacting drugs
MLSA1
07
Biomarkers
TRPML1 expression levels in glioblastoma as a prognostic marker
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