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MOSMO is a membrane tetraspan protein that functions as a negative regulator of the Hedgehog signaling pathway by promoting the internalization and degradation of the smoothened (SMO) transducer, consequently downregulating pathway activation. It plays essential roles in embryonic development, particularly in craniofacial and neural tube formation, with deficiency or inactivation leading to congenital malformations. MOSMO is distinct from the Smoothened receptor itself; rather, it modulates SMO activity and thereby influences Hedgehog signaling output[5][7].
Drugs targeting the Hedgehog pathway (e.g., Smoothened inhibitors) might act indirectly on the pathway influenced by MOSMO, but no drugs directly targeting MOSMO have been described. MOSMO acts by promoting internalization and degradation of Smoothened, thereby reducing pathway activation[5].
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