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Target intelligence / Profile preview
Survival motor neuron 2 protein (SMN2)
- Target
- SMN2
- Molecular classification
- RNA-binding protein, Spliceosome component, Other
01
Overview
Survival motor neuron 2 protein is an essential component for RNA processing machinery integrity and neuronal health. Its deficiency underlies spinal muscular atrophy due to impaired pre-mRNA splicing machinery assembly—a defect that can be therapeutically targeted by modulating alternative splicing from its encoding gene (*SMN2*).
- Other names
- BCD541C-BCD541GEMIN1TDRD16B
02
Mechanism of action
Enhances correct splicing from *SMN2*, increasing production of functional full-length SMN protein
03
Biological functions
snRNP biogenesissnoRNP biogenesisPre-mRNA splicingRNA metabolismDNA repairTranscription regulationTranslation controlMitochondrial homeostasisEndocytosis/cytoskeleton dynamicsUbiquitination/autophagyMacromolecular traffickingStress granule formationAxon/dendrite outgrowth
04
Disease associations
Spinal muscular atrophy (SMA)Male infertilityInclusion body myositisAmyotrophic lateral sclerosis (ALS)Osteoarthritis
05
Interacting drugs
Branaplam
06
Biomarkers
Total cellular levels of functional full-length SMN
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