Antisense oligonucleotide

An antisense oligonucleotide (ASO) is a short, synthetic piece of DNA or RNA designed to bind specifically to a targeted messenger RNA (mRNA) in cells. By attaching to this mRNA, the ASO can block the production of a particular protein. This approach allows for precise control over gene expression, which can be useful in treating certain genetic disorders, cancers, or viral infections. Essentially, ASOs act as molecular "brakes," stopping unwanted proteins from being produced, offering a targeted therapeutic strategy with minimized effects on other genes.