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The mapping of the human genome unveiled an abundance of potential molecular targets. The challenge for the pharmaceutical industry is to gain valuable insight into the role these targets play in the body and their viability as drug targets. Ultimately, scientists seek to understand which genes are directly involved in disease pathways in order to develop drugs specific to key gene targets in a timely manner. This overall task is referred to as functional genomics. Traditional drug discovery screening methods are time-consuming, often requiring years to identify lead drug candidates.

Antisense technology offers an efficient solution to this challenge. Antisense inhibitors, or drugs, can be used to identify what a gene does and whether a specific gene is a good drug target. The characteristics of antisense that make it an ideal tool for functional genomics, are outlined as follows:

• Highly Specific – inhibits the expression of one gene

• Universal – able to produce inhibitors to any gene; the DNA sequence is the only information needed

• Versatile – works in cultured cells, animals and man

• Verifiable – able to validate every experiment through a rigorous set of controls

• Rapid – able to produce inhibitors within a week; scale up for animal studies in a month

• Pharmacologically Predictive – provides drug developers with data on the temporary inhibition of a target protein and the precise effect of a drug, because antisense inhibitors are drugs

The use of antisense technology to identify the role of genes is simply the first step in Isis’ drug discovery process.