IMPACT OF THE HUMAN GENOME PROJECT ON THE DESIGN OF NEW MEDICINES
DNA is the pharmacological target of many of the drugs that are currently in clinical use or in advanced clinical trials. Targeting DNA to regulate cell functions by modulating gene expression or by interfering with replication seems logical, intuitively appealing and conceptually straightforward. With the new wealth of information provided by the Human Genome Project, it was expected that this information would lead to a quantum leap in the development of DNA-binding pharmaceuticals since this information should facilitate the elucidation of the roles that different genes play in important pathological processes. Pharmacology is thought to be a major potential beneficiary from the completion of the first draft of the Human Genome Project since in the post-genomic era, it seems quite plausible that in the near future it may be possible to combine genomics and pharmacology in order to administer specific individual treatments based on the individual's genotype. Although in the 'post genomic' era the fields of functional genomics and proteomics have been receiving increasing attention, many researchers feel that it is more efficient to treat a disease by designing drugs that act at the DNA or messenger RNA level rather than at the protein level.
The Human Genome Project
Begun formally in 1990, the U.S. Human Genome Project was a 13-year effort coordinated by the U.S. Department of Energy and the National Institutes of Health. The project originally was planned to last 15 years, but rapid technological advances accelerated the completion date to 2003. Project goals were to
• identify all the approximately 20,000-25,000 genes in human DNA,
• determine the sequences of the 3 billion chemical base pairs that make up human DNA,
• store this information in databases,
• improve tools for data analysis,
• transfer related technologies to the private sector, and
• address the ethical,...