Designing new drugs through DNA

The human genome project will sequence the human genome within the next 2 years. Tools have been created that allow the use of genetics to study inherited diseases in humans where the genetic component may be complex. Most human genes have been identified and tools exist to use this information to search for abnormal expression of genes in diseased tissues. Many medically important pathogens have already had their genomes sequenced.

Can this deluge of information be used effectively to design new drugs? Will it help in producing new therapies. Where are the bottlenecks in pharmacologic development and will the genome information revolution lift some of these blocks? This article addresses some of these issues and seeks to demonstrate that the genome project will inject an enthusiasm into the development of new pharmacologic agents against a large range of hitherto unknown target molecules.

Pharmaceutical development is target limited. This is especially true in the microbiological arena where there have been no new targets used for the past decade or more. New genes will be uncovered that play a role in disease processes. These may not be the causative genes but will modulate the course of disease. These new target proteins will be identified either as a result of the large scale sequencing efforts, now underway in a small number of large laboratories in the USA and Europe or will be found as a result of applying this knowledge to find genes that are inappropriately expressed in diseased tissue.