Antisense oligonucleotides (ASOs) are a single-stranded DNA or RNA sequence consisting of 15-25 nucleotides paired with a target gene. It can regulate gene expression by blocking the transcription or translation of target genes. Antisense oligonucleotides have been widely used in gene therapy due to their advantages of high specificity, high efficiency, and low toxicity.
In recent years, ASOs have often been used to develop gene-targeted therapy drugs. Natural ASOs are easily degraded in vivo with low specificity and toxic side effects. However, the ASO drugs usually have specific modifying groups to enhance the stability of oligonucleotides in vivo, improve their specificity, and reduce their toxic side effects.
Synbio Technologies has established production workshops that meet the quality management requirements of ISO 9001 and ISO 13485, with a standard production process and excellent synthesis & purification technologies. Our synthetic ASOs strictly comply with QC testing standards. HPLC purity detection is used to ensure the high quality output of all our ASO products.
Antisense DNA oligonucleotides (ASOs) have multiple applications in the field of molecular biology, with a particular focus on studying and identifying RNAi phenotypes. Here are some of the most common applications of ASOs:
- Gene expression knockdown: ASOs can be designed to target specific messenger RNA (mRNA) transcripts and prevent their translation into protein. This can be achieved by disrupting the binding of ribosomes to the mRNA, cleaving the mRNA, or recruiting RNase H to degrade the mRNA. ASOs can be used to study the function of a gene by observing the effects of its knockdown.
- Alternative splicing regulation: ASOs can influence the splicing of pre-mRNA transcripts and result in the alteration of gene expression. By binding to intronic or exonic sequences, ASOs can modify the splicing machinery’s binding affinity and promote or hinder inclusion or exclusion of exons.
- RNA editing: ASOs can be designed to recruit adenosine deaminases to facilitate RNA editing. The ASO sequences can bind to double-stranded RNA, exposing specific adenosine residues to deamination and resulting in the conversion of adenosine to inosine, which is translated as guanosine. This approach has been applied to study RNA editing in disease models.
- Antisense therapy: ASOs can be designed to target specific disease-causing mRNAs, such as those coding for oncogenic proteins, in order to inhibit their translation and reduce pathological effects. This approach has been used for diseases like spinal muscular atrophy and hereditary angioedema, with some ASOs undergoing clinical trials.
- Aptamer-mediated delivery: ASO sequences can be used as a scaffold for attaching aptamers, which are nucleic acid sequences that bind to specific targets with high affinity and specificity. The resulting conjugates can be used for targeted delivery of drugs or other molecules to specific cells or tissues.
ASOs are powerful tools for investigating gene function, regulating gene expression, and developing new therapies for genetic and other diseases. Their versatility and specificity make them a valuable resource for molecular biologists and clinicians alike.