Antisense technology is a method of controlling gene expression in a cell by using synthetic antisense oligonucleotides. These oligonucleotides are short chemically modified strands of DNA or RNA that are complementary to a specific mRNA sequence. When they bind to the mRNA, they can inhibit or downregulate the production of the target protein. Antisense technology can be used to treat a variety of diseases by targeting RNA. There are two possible mechanisms for an antisense effect: the antisense strategy, which targets mRNA, and the antigene strategy, which targets double-stranded DNA or genes situated in the nucleus. The antisense strategy is well established with several examples of in vitro and in vivo applications.
Some key terms related to antisense technology include:
- Antisense oligonucleotide (ASO): A short chemically modified strand of DNA or RNA that is complementary to a specific mRNA sequence.
- RNase H1: An endoribonuclease that specifically cleaves the RNA strand present in a DNA-RNA heteroduplex and is the cellular enzyme used by antisense oligonucleotides to trigger specific degradation of complementary RNA.
- Exon skipping: A process by which an exon that is normally present in mature mRNA is omitted during splicing, and thus is not present in mature mRNA. Antisense oligonucleotides can be designed to promote exon skipping with the goal of producing a shorter, but still functional, protein.
Antisense technology is a promising tool for controlling gene expression and treating diseases. It has great potential for cancer therapy.
