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Target intelligence / Profile preview
Survival of Motor Neuron 1 Protein (SMN1)
- Target
- SMN1
- Molecular classification
- RNA-binding protein, Spliceosome component, Other
01
Overview
The survival of motor neuron 1 protein (SMN1) is a ubiquitously expressed protein essential for the maintenance and function of motor neurons. It plays a crucial role in assembling snRNPs, which are critical components of the spliceosome. Mutations or deletions in both copies of the SMN1 gene cause spinal muscular atrophy (SMA), a genetic disorder characterized by progressive muscle weakness.
- Other names
- SMNSurvival Motor Neuron
02
Mechanism of action
Enhancement of SMN protein levels
03
Biological functions
snRNP assemblymRNA processingMitochondrial homeostasisEndocytosisCytoskeleton regulationAxonal transport
04
Disease associations
Spinal Muscular AtrophyNeurodegenerative disease
05
Safety considerations
Off-target effects of SMN-enhancing therapies
06
Biomarkers
SMN protein levels in blood or tissueSMN2 copy number
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