Review Article
Variables and Strategies in Development of Therapeutic Post-Transcriptional Gene Silencing Agents
Table 1
Comparison of the properties of antisense, ribozyme, and RNAi.
| | Property | Antisense | Ribozyme | siRNA/shRNA/miRNA |
| | Size | Small (15–20 nt) | Small (42–60 nt) | Small (19–22 nt)* | | Crystal structure | No | Yes | Yes (RISC) | | Mechanism of action | Known | Known | Known | | Independence cell metabolism | No | Yes | No | | Specificity | Moderate | High | Poor to moderate | | Saturable | No | No | Yes | | Cellular compartment | Cytoplasm | Nucleus/cytoplasm | Cytoplasm | | Dependence on target structure | Yes | Yes | Yes | | Proven in vivo | Yes | Yes | Yes |
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*On one strand engaged in the RISC complex after cellular processing.
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