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Target intelligence / Profile preview
Ankyrin repeat and MYND domain-containing protein 2 (ANKMY2)
- Target
- ANKMY2
- Molecular classification
- Other (cytosolic trafficking protein), Ankyrin repeat family protein, MYND domain-containing protein
01
Overview
Ankyrin repeat and MYND domain-containing protein 2 (ANKMY2) is a cytosolic trafficking protein that regulates the maturation and ciliary localization of adenylyl cyclases, thereby repressing Hedgehog pathway activity during development[1][2][4]. Loss of ANKMY2 results in severe neural tube ventralization and embryonic lethality due to hyperactivation of the Hedgehog pathway independently of Smoothened, underscoring its critical role in cilia-dependent signal repression and morphogenetic patterning[2][4]. ANKMY2 is a member of the ankyrin-repeat and MYND domain protein family, which generally mediates protein-protein interactions and cellular signaling[1][3]. While its function as a direct therapeutic target is not established, ANKMY2 represents a key node in understanding ciliary signaling, developmental disorders, and potentially cancer biology.
- Other names
- Ankmy2
02
Mechanism of action
Drugs would act by modulating adenylyl cyclase maturation or trafficking, consequently altering Hedgehog pathway activity
03
Biological functions
Trafficking of adenylyl cyclases to ciliaRegulation of cilia-dependent signalingRepression of Hedgehog pathway signalingPromotion of Gli-repressor formationInvolvement in morphogenetic patterning during embryonic development
04
Disease associations
Cancer (suggested by involvement in Hedgehog pathway regulation, although direct evidence in human cancer is limited)Developmental disorders (neural tube defects due to Hedgehog pathway dysregulation)Other (potential ciliopathy due to defective signaling protein trafficking)
05
Safety considerations
Embryonic lethality observed in knockout modelsSevere developmental defects (e.g., open neural tube, abnormal neural patterning)Potential for off-target effects due to cilia and signaling disruption
06
Interacting drugs
None directly reported; clinical targeting is experimental and not established
07
Biomarkers
Hedgehog pathway activation markers (e.g., ventral neuroprogenitor markers, Gli2 and Gli3 repressors, neural tube closure defects)
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