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Target intelligence / Profile preview
Survival motor neuron domain-containing protein 1 (SMNDC1)
- Target
- SMNDC1
- Molecular classification
- RNA-binding protein, Splicing factor, Tudor domain protein, Member of spliceosome complex
01
Overview
Survival motor neuron domain-containing protein 1 (SMNDC1) is an essential nuclear RNA-binding protein and splicing factor that recognizes dimethylated arginines via its Tudor domain, localizes to phase-separated nuclear speckles, and regulates spliceosome assembly and pre-mRNA splicing. It plays major roles in cell identity, proliferation, and gene expression, with important implications in cancer, diabetes, and muscular atrophy. Inhibitors targeting its Tudor domain disrupt its molecular function, offering potential for therapeutic intervention but carrying risks due to its essentiality for most cell types
- Other names
- Survival of motor neuron-related-splicing factor 30SPF30SMNRTDRD16C30 kDa splicing factor SMNrpSMN-related proteinsplicing factor 30survival of motor neuron-relatedtudor domain-containing 16C
02
Mechanism of action
Inhibition of dimethylarginine binding pocket of Tudor domain disrupts protein-protein interactions, subcellular localization, and spliceosome assembly, leading to downstream splicing changes affecting gene expression
03
Biological functions
Pre-mRNA splicingGene expression regulationRNA processingPhase separationNuclear compartmentalizationCell proliferation
04
Disease associations
Cancer (notably hepatocellular carcinoma)DiabetesMuscular atrophySpinal muscular atrophy (by association/paralog)Potential roles in endocrine pancreas cell fate
05
Safety considerations
Essential gene: complete loss impairs cell viability in most cell typesPossible broad impact on splicing and gene expression when inhibited, raising concerns for on-target and off-target effects in therapy
06
Interacting drugs
Small molecule Tudor domain inhibitors (identified but not clinically approved)
07
Biomarkers
High SMNDC1 expression correlates with poor survival in liver cancerKnock-down linked to changes in insulin expression and PDX1 as markers in pancreatic alpha cells
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