Gene therapy drug discovery

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Having examined the principal features of combinatorial chemistry as an ongoing business concern, it is instructive and useful to compare and contrast the field with other major platform biotechnologies that are shaping modern drug discovery and development. In particular, combinatorial chemistry will be compared with gene therapy and also with the business that has emerged as a result of the invention of the Polymerase Chain Reaction (PCR). 

Like combinatorial chemistry, gene therapy is a platform biotechnology that relies on several key technical contributions that have arisen in several academic and company laboratories. By contrast, PCR is a single invention, unlike combinatorial chemistry or gene therapy, but, like the other two platforms used as examples here, it has also been able to completely revolutionize the process of drug discovery. 

The study of the proteome of the recombinant adenovirus type 5 vectors demonstrated an important apphcation of separation techniques in combination with MS methods in the drug discovery process. With completely sequenced adenovirus genome available, this approach provides a chemically well-dehned method of characterization of structural proteins of recombinant adenoviral vectors. The information of protein MWs, tryptic peptide mass mapping, and sequence tags of tryptic peptides derived from HPLC/MS resulted in the identification of 17 adenoviral proteins/polypeptides in the purified virion. The rapid and accurate identification of viral proteins from recombinant adenoviruses in this study is significant since it provides direct evidence of the maturation stage of adenoviruses, which is closely related to viral infectivity and efficacy in gene therapy. 

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