Overview
Through ASO (antisense oligonucleotide) or siRNA (small interfering RNA)-mediated gene silencing technology, target gene expression is suppressed at transcriptional or translational levels, validating the functional importance of targets and their feasibility as drug targets.
Technology Comparison
| Feature | siRNA | ASO |
|---|---|---|
| Site of action | Cytoplasm (mRNA degradation) | Nucleus (splice blocking/RNase H degradation) |
| Duration | Days to ~1 week | Weeks to months |
| Delivery | Lipofection/electroporation/GalNAc | Naked delivery/Gapmer/GalNAc |
| Off-target risk | Seed-dependent off-target | Hybridization-dependent off-target |
Validation Metrics
- mRNA level knockdown (qPCR verification, typically > 70% knockdown efficiency)
- Protein level reduction (Western Blot / flow cytometry verification)
- Functional phenotypic changes (proliferation, apoptosis, migration, etc.)