Target intelligence / Profile preview

Anoctamin-3 (ANO3)

Target
ANO3
Molecular classification
Ion channel (TMEM16/anoctamin family), Membrane protein, Phospholipid scramblase
01

Overview

Anoctamin-3 (ANO3) is a transmembrane protein encoded by the ANO3 gene on human chromosome 11, belonging to the TMEM16/anoctamin family, whose members often function as ion channels or phospholipid scramblases[1][2][3][4][6]. While originally described as a putative calcium-activated chloride channel, current evidence suggests ANO3 acts primarily as a calcium-dependent phospholipid scramblase and modulator of potassium channel activity rather than a classical chloride channel[2][3]. ANO3 exhibits high expression in neuronal tissues—especially in basal ganglia, striatum, cortex, hippocampus, and dorsal root ganglia—where it regulates neuronal excitability and pain signaling, partly by facilitating the function of sodium-activated potassium channels (KCNT1/Slack)[2][3]. Human mutations in ANO3 are causative for autosomal dominant craniocervical dystonia (DYT23), characterized by involuntary movements and tremor of the face, head, and neck[1][2][3]. ANO3 function may also have relevance to neurodegenerative and pain disorders, but no drugs are currently known to selectively target ANO3, and its involvement as a direct therapeutic target is still under investigation[2][3].

Other names
Transmembrane protein 16CTMEM16CC11orf25DYT23DYT24Anoctamin 3anoctamin 3dystonia 23
02

Biological functions

Modulation of potassium channel activityPhospholipid scramblingRegulation of neuronal excitabilityRegulation of pain signalingRegulation of ER-dependent calcium signaling
03

Disease associations

Neurodegenerative disease (Dystonia, DYT23)Pain disordersPotential role in late-onset Alzheimer's disease
04

Safety considerations

Mutations associated with movement disorders (craniocervical dystonia, tremor, myoclonic jerks)loss of function in animal models linked to altered pain sensitivity and neuronal hyperexcitability[1][2][3]
05

Biomarkers

Mutations in ANO3 as a genetic marker for craniocervical dystonia (DYT23)high expression in certain brain regions may have biomarker potential for disease association[1][2][3]

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