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Target intelligence / Profile preview
Survival of Motor Neuron 2 (SMN2)
- Target
- SMN2
- Molecular classification
- RNA-binding protein, Transcription factor
01
Overview
Survival of motor neuron 2 (SMN2) is a gene that encodes the survival motor neuron (SMN) protein, which is essential for motor neuron maintenance and function. SMN2 primarily produces a truncated, non-functional protein due to exon 7 skipping. However, a small percentage of transcripts produce full-length SMN protein, and increased SMN2 copy number can partially compensate for the loss of SMN1 function in spinal muscular atrophy. Therefore, it is a therapeutic target.
- Other names
- BCD541C-BCD541GEMIN1TDRD16BSMNC
02
Mechanism of action
Therapeutics target increased expression or correct splicing from existing SMN2-derived transcripts
03
Biological functions
snRNP assemblySpliceosome formationMotor neuron maintenanceRNA splicingCytoskeletal dynamicsMitochondrial homeostasisEndocytosisUbiquitinationAutophagyRNA transport
04
Disease associations
Spinal Muscular AtrophyNeurodegenerative disease
05
Safety considerations
Therapeutic strategies must carefully balance increased SMN protein production with potential off-target effects.
06
Biomarkers
SMN2 copy numberFull-length SMN protein levels
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