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Target intelligence / Profile preview
Adaptor protein complex 4 subunit mu-1 (AP4M1)
- Target
- AP4M1
- Molecular classification
- Vesicle coat component (adaptor protein), Adaptor complexes medium subunits family
01
Overview
Adaptor protein complex 4 subunit mu-1 (AP4M1) is one of four components forming the heterotetrameric adaptor protein complex AP-4. This medium-sized subunit plays an essential role in recognizing and binding tyrosine-based sorting signals on cargo proteins within the cytoplasmic domain. The AP-4 complex mediates vesicular trafficking by facilitating transport from the trans-Golgi network to endosomes and lysosomes. In neurons specifically, it regulates somatodendritic distribution of key membrane proteins such as AMPA-type glutamate receptors. Mutations in AP4M1 disrupt these processes and cause autosomal recessive hereditary spastic paraplegia type 50 (SPG50), characterized by progressive lower limb spasticity and intellectual disability due to impaired brain development and function.
- Other names
- AP4M1CPSQ3MU-4MU-ARP2SPG50adaptor related protein complex 4 mu 1 subunitadaptor related protein complex 4 subunit mu 1
02
Biological functions
Recognition and sorting of cargo proteins with tyrosine-based motifs from the trans-Golgi network to the endosomal–lysosomal systemRegulation of somatodendritic-specific distribution of cargo proteins including AMPA receptors in neurons
03
Disease associations
Neurodegenerative disease (specifically hereditary spastic paraplegia type 50 / SPG50)
04
Safety considerations
No notable safety concerns or therapeutic challenges reported specific to targeting this molecule; however, loss-of-function mutations cause severe neurodevelopmental disorders (spastic paraplegia)
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