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Target intelligence / Profile preview
Fragile-X multifunctional RNA-binding protein (FMRP)
- Target
- FMRP
- Molecular classification
- RNA-binding protein
01
Overview
The Fragile-X multifunctional RNA-binding protein (FMRP) is the protein product of the FMR1 gene. It is a highly conserved RNA-binding protein that regulates translation, mRNA transport and stability, and participates in DNA damage response. Mutations in FMR1 leading to loss or dysfunction of FMRP cause Fragile X Syndrome, a common form of inherited intellectual disability.
- Other names
- Fragile X Mental Retardation ProteinFragile X Messenger Ribonucleoprotein 1Synaptic functional regulator FMR1
02
Biological functions
Translation regulationmRNA transportmRNA stabilizationDNA damage responseSynaptic plasticityNeuronal development
03
Disease associations
Fragile X SyndromeIntellectual disabilityNeurodevelopmental disorders
04
Safety considerations
Potential for off-target effects due to widespread expressionChallenges in specifically targeting FMRP in the brain
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