Tutorial Antisense Technologies

IDTutorial: Antisense Technologies Introduction Antisense technologies are a suite of techniques that, together, form a very powerful weapon for studying gene function (functional genomics) and for discovering new and more specific treatments of diseases in humans, animals, and plants (antisense therapeutics). A conventional definition of antisense refers to the laboratory manipulation and/or modification of DNA or RNA so that its components (nucleotides) form a complementary copy of normal, or “sense,” messenger RNA (mRNA). The binding, or hybridization, of antisense nucleic acid sequences to a specific mRNA target will, through a number of different mechanisms, interrupt normal cellular processing of the genetic message of a gene. This interruption, sometimes referred to as “knock-down” or “knock-out” depending upon whether or not the message is either partially or completely eliminated, allows researchers to determine the function of that gene. In this review, a survey of the agents employed in antisense technologies will be presented along with a discussion of the various mechanisms they employ to achieve the goal of reducing or eliminating normal processing of a gene of interest. The focus will be on those techniques that employ oligonucleotides composed of both modified and unmodified DNA and/or RNA nucleotides. Another major antisense technology, called “RNA Interference”, or RNAi, will be presented in more detail in another ...