Fragile X messenger ribonucleoprotein 1 (FMRP)

Fragile X messenger ribonucleoprotein 1 (FMRP) is an RNA-binding protein encoded by the FMR1 gene on the X chromosome, with critical roles in synaptic function, neuronal development, and cognitive processes. FMRP regulates the translation, transport, and localization of mRNAs, especially in neurons, where it is essential for normal synaptic plasticity. Loss or mutation of FMR1 causes fragile X syndrome, the most common inherited cause of intellectual disability, and is associated with a spectrum of neurodevelopmental and neurodegenerative disorders, including autism and premature ovarian failure. FMRP exerts translational repression on key targets involved in synaptic structure and function, influencing local protein synthesis in response to synaptic activity, with its activity tightly regulated by metabotropic glutamate receptor (mGluR) signaling.

Other names

FMR1Fragile X mental retardation proteinfragile X messenger RNA binding protein

Not applicable for direct targeting; when targeted indirectly, drugs modulate synaptic protein synthesis via mGluR signaling pathways affected by loss of FMRP function