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Target intelligence / Profile preview
Transient Receptor Potential Cation Channel Mucolipin Subfamily Member 1 (TRPML1)
- Target
- TRPML1
- Molecular classification
- Ion channel, TRP channel, Cation channel
01
Overview
TRPML1 is a non-selective, pH-regulated cation channel primarily located on late endosomes and lysosomes. It is involved in lysosomal calcium release, membrane trafficking, autophagy, and other cellular processes. Loss-of-function mutations in MCOLN1, the gene encoding TRPML1, cause mucolipidosis type IV (MLIV).
- Other names
- MCOLN1
02
Mechanism of action
Modulation of TRPML1 channel activity (activation/inhibition)
03
Biological functions
Lysosomal calcium signalingMembrane traffickingAutophagyExocytosisLipid traffickingIon transport
04
Disease associations
Mucolipidosis type IVNeurodegenerative diseaseLysosomal storage disorder
05
Safety considerations
Potential for off-target effects on other TRP channelsComplex regulation of channel activity
06
Interacting drugs
ML-SA1
4 more in the full profile.
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