Proteins play a central role in virtually every aspect of human metabolism. Traditional drugs are designed to interact with protein molecules throughout the body that support or cause diseases. Antisense drugs are designed to inhibit the production of disease-causing proteins. They can be designed to treat a wide range of diseases including infectious, inflammatory and cardiovascular diseases and cancer and have the potential to be more selective and, as a result, more effective and less toxic than traditional drugs. The information necessary to produce proteins in cells is contained in genes.
Specific genes contain information to produce specific proteins.
The information required for the human body to produce all proteins is contained in the human genome and its collection of more than 100,000 genes. Genes are made up of DNA which contains information about when and how much of which protein to produce, depending upon what function is to be performed. During transcription of information from DNA into mRNA, the two complementary strands of the DNA partly uncoil. Then, mRNA migrates into the cell where other cellular structures called ribosomes read the encoded information, its mRNA's base sequence, and in so doing, string together amino acids to form a specific protein. Antisense drugs have the potential to be much more selective or specific than traditional drugs, and therefore more effective, because they bind to mRNA targets at multiple points of interaction at a single receptor site.