ASO synthesis

ASO, also known as antisense oligonucleotides, refers to short, artificially synthesized oligonucleotides, typically 15-25nt, that can complement the RNA/DNA regions of target genes, thereby preventing their transcription and translation. ASO directly targets specific RNA through the principle of base complementarity, affecting the expression of target proteins. In the field of small nucleic acid drugs, this characteristic can avoid the limitation of non drug targets faced by traditional small molecule drugs and antibody drugs. The mechanism of action of ASO can be mainly divided into two types: one is to directly participate in RNA degradation (see Figure a for the mechanism of action), and the other is to inhibit RNA expression through steric hindrance (see Figures b, c, d, e for the mechanism of action).

Mechanism of ASO action

ASO can use various chemical modifications to improve stability, avoid degradation by nucleases, reduce immunogenicity, etc., mainly including modifications to ribose, phosphate backbone, bases, and nucleic acid chain ends. At the same time, ASO can also use delivery carriers such as liposomes, GalNAc modifications, linked antibodies, linked peptides, copolymers, etc. The most approved nucleic acid drug currently is ASO, with 11 drugs approved for market. The approved ASO drugs are as follows:

The common ASO modifications are as follows:

Biosyntech Biotechnology can provide various modified ASO products, with commonly used modification types as follows: