The impact of genomics and related technologies upon drug discovery

The impact of genomics and related technologies upon drug discovery 

Microbe Genome size (MB) Microbe Genome size (MB) 

The assignment of function to the products of sequenced genes can be pursued via various approaches, including  

The gene neighbourhood method is yet another computation-based method. It depends upon the observation that if two genes are consistently found side by side in the genome of several different organisms, they are likely to be functionally linked. 

Knock-out animal studies, in contrast to the above methods, are dependent upon phenotype observation. The approach entails the generation and study of mice in which a specific gene has been deleted. Phenotypic studies can sometimes yield clues as to the function of the gene knocked out. 

In other cases, the widespread application of a biopharmaceutical may be hindered by the occurrence of relatively toxic side effects (as is the case with tumour necrosis factor a (TNF-a, Chapter 9). Finally, some biomolecules have been discovered and purified because of a characteristic biological activity that, subsequently, was found not to be the molecule s primary biological activity. TNF-a again serves as an example. It was first noted because of its cytotoxic effects on some cancer cell types in vitro. Subsequently, trials assessing its therapeutic application in cancer proved disappointing due not only to its toxic side effects, but also to its moderate, at best, cytotoxic effect on many cancer cell types in vivo. TNF s major biological activity in vivo is now known to be as a regulator of the inflammatory response. 

In summary, the discovery of biopharmaceuticals, in most cases, merely relates to the logical application of our rapidly increasing knowledge of the biochemical basis of how the body functions. These substances could be accurately described as being the body s own pharmaceuticals. Moreover, rapidly expanding areas of research, such as genomics and proteomics, will likely hasten the discovery of many more such products, as discussed below. 

From a drug discovery/development prospective, the significance of genome data is that they provide full sequence information of every protein the organism can produce. This should result in 

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THE IMPACT OF GENOMICS AND RELATED TECHNOLOGIES UPON DRUG DISCOVERY... 

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