Nucleic Acid Sequence Design

Nucleic Acid Sequence Design (Figure 1):

siRNA (small interfering RNA) achieves gene silencing by specifically binding and cleaving target mRNA through RNA interference mechanism. It binds to Ago proteins and other proteins in the cytoplasm to form RISC (RNA-induced silencing complex) and performs its function with precision. saRNA (small activating RNA) is similar in structure to siRNA, yet it can activate gene expression by acting on a promoter or a specific regulatory element.

ASO (Antisense Oligonucleotide) is a single-stranded oligonucleotide molecule, usually containing 15-25 nucleotides, which binds to the target RNA through base complementation and interferes with its shearing or translation. According to the mechanism of action, they can be classified into RNase H-dependent and spatial site-blocking types.

Advantages of HitGen oligonucleotide design:

1. Scoring system: a neural network-based ‘2nd-gen AI/ML’ algorithmic platform that cross-analyses siRNA scoring and active interference data to improve the accuracy of targeting efficiency prediction models (Figure 2).

2. Off-target prediction algorithm: integrating sequence information and secondary structure to assess potential off-target risk and provide early warning of high-risk off-target sequences (Figure 3).

Figure 1：Workflow of design

Figure 2. Correlation of siRNA scores and KD effects   

Figure 3. transcriptome expression of siRNA sequences with different on-target scores