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Survival of Motor Neuron 2 Protein (SMN2)
- Target
- SMN2
- Molecular classification
- RNA-binding protein, Spliceosomal protein
01
Overview
The survival of motor neuron 2 protein (SMN), encoded by the SMN2 gene, is a critical protein found in all animal cells, particularly in the spinal cord. It is essential for the maintenance and function of motor neurons. Its primary role is in the assembly of spliceosomal snRNPs required for pre-mRNA splicing. Deficiency or dysfunction leads to spinal muscular atrophy (SMA).
- Other names
- SMNBCD541C-BCD541GEMIN1SMNCTDRD16B
02
Mechanism of action
Risdiplam and Nusinersen enhance SMN2 exon 7 inclusion, increasing functional SMN protein production. Onasemnogene abeparvovec delivers a functional SMN1 gene.
03
Biological functions
snRNP assemblypre-mRNA splicingTranscriptional regulationTelomerase regenerationCellular traffickingMitochondrial homeostasisEndocytosisCytoskeleton regulationUbiquitinationAutophagyRNA transport
04
Disease associations
Spinal Muscular Atrophy (SMA)
05
Safety considerations
Nusinersen: Post-lumbar puncture syndrome, bleeding risks, thrombocytopenia, renal toxicityRisdiplam: Potential developmental toxicityOnasemnogene abeparvovec: Elevated liver enzymes, acute liver failure, thrombocytopenia, thrombotic microangiopathy
06
Interacting drugs
Risdiplam
2 more in the full profile.
07
Biomarkers
SMN protein levelsSMN2 copy number
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