Antisense Oligonucleotides as Research Tools

The use of antisense oligonucleotides as both research tools and therapeutic molecules has emerged as a powerful alternative to small molecule inhibitors. Antisense oligonucleotides are short pieces of chemically modified DNA designed to hybridize to specific mRNA sequences present in the target gene. The oligonucleotide interaction with the targeted mRNA can lead to inhibition in the translation of the protein encoded by the targeted transcript through a variety of reasonably well-characterized mechanisms (1 –3)