With this core competency, the pharmaceutical industry is likely to be a primary beneficiary of the Genomic Era. Proteins are natural targets for interventions in the treatment of disease. Alternatively, proteins can be artificially manufactured and introduced into the body, often to replace a protein that is not present in sufficient quantity. Frequently, individual proteins operate as parts of larger systems that affect a given function, making for a plurality of potential targets for intervention associated with a given system. Whether the means is via a small molecule drug or introduced protein, the game of therapeutic development is to identify the proteins involved in the disease process in the cell and determine a means to affect their function.
Gene therapy is the ultimate endgame of genomics.
In gene therapy, defective genes are simply identified and repaired. For example, in diabetes the genes responsible would be fixed, obviating the need for insulin and glucose monitoring. Success in gene therapy to date has been mixed. Although a few gene therapy treatments could be marketed during the 2000 decade, widespread use of gene therapy probably remains several decades away.