Well designed siRNAs are potent and specific modulators of gene expression and have immense potential in therapeutic gene silencing. Very successful design rules and algorithms have been established based on performance of hundreds of siRNA and miRNAs (miRNAs are naturally occurring and structurally resemble siRNA and use a similar silencing complex).
1. Effective cell membrane permeation.
2. Targeted delivery.
3. Nuclease resistance confers increased half-life.
4. Sustained gene silencing with increased duplex stability.
5. Minimal off-target seeding and silencing
6. Low dosage. Minimal Toxicity