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Target intelligence / Profile preview
Survival Motor Neuron Protein (SMN)
- Target
- SMN
- Molecular classification
- RNA-binding protein, Spliceosome component, Housekeeping protein
01
Overview
The survival motor neuron (SMN) protein is a ubiquitously expressed, multifunctional protein essential for the survival of all animal cells. It plays critical roles in snRNP and snoRNP biogenesis, pre-mRNA splicing, and a variety of other cellular processes. Deficiency in SMN protein, primarily due to mutations in the SMN1 gene, is the cause of spinal muscular atrophy (SMA), a severe neuromuscular disorder.
- Other names
- SMN1SMN
02
Mechanism of action
SMN enhancing therapies aim to increase the production of functional SMN protein, often by modulating SMN2 splicing or increasing SMN protein stability.
03
Biological functions
snRNP biogenesissnoRNP biogenesisPre-mRNA splicingRNA metabolismmRNA traffickingCytoskeletal dynamicsEndocytosisAutophagyMitochondrial functionDNA repairSelenoprotein synthesisMacromolecular traffickingStress granule formationCell signaling
04
Disease associations
Spinal Muscular Atrophy (SMA)Male infertilityInclusion body myositisAmyotrophic Lateral SclerosisOsteoarthritis
05
Safety considerations
Potential for off-target effects due to widespread expressionDosage optimization to avoid excessive SMN production
06
Biomarkers
SMN protein levels in blood or tissueMotor neuron survivalMuscle function assessments
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