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The Potential of the Humane Genome Project

Posted by Neal on September 22, 2001, at 1:25:35

The future of parmacology may be based on our genes. The Human Genome Project was established in 1990. The goal of the Project is to work out the complete DNA sequence of the human genome, in essence, to describe all 80-100,000 human genes. The project is now complete. Biology, medicine, and industry have forged a powerful alliance in one of the biggest initiatives ever undertaken by science. It will reframe our concepts of disease and therapy forever.

One potential outcome from the Human Genome Project could be the ultimate achievement of specific medical therapy, manipulating of our genes, the very stuff of life itself. A major driving force of the project is the identification of genes that predispose us to disease in order that they may be "cured" by gene thrapy; that is, physically replace the defective gene witha normal one.

But promises of replacing damaged genes have been with us for more than a decade without any unequivical case of successful gene therapy. Moreover, there now exists a growing divergence of opinion among scientists as to whether it is possible that the function a a single gene can either cause of cure a disease. Indeed, it is very difficult to predict the role of genes in any complex biological function.

Huntington's disease, for example, is a relatively rare brain disease associated with symptoms of depression, personality change, impaired memory, and abnormal involuntary morvements. It results from the inheritance of a single abnormal gene. An important feature of Huntington's disease is that it does not strike until middle age, which illustrates the critical point that a particular gene, although transmitted at conception, may exhibit no detectable influence for many years. Moreover the abnormal protein that the gene encodes is rarely found in the neurons most vulnerable to the disease. Why the same gene can be present in many different kinds of cells, but only some be affected is not known. The interaction of some other gene(s) must be required.

Yet the identification of every single gene and, by extrapolation, the structure of the proteins they encode, has great potential for the pharmacologist, because it will allow the development of drugs of unique specificity on a scale never before imagined. The flow of gene information from the Human Genome Project is so massive that our existing knowledge base will have to be buttressed by the emerging science of bioinformatics and combinatorial chemistry.

These techniques allow us to sift through databases to compare, contrast, and continually modify a basic chemical structure until the perfect fit for its targe receptor is found. The limits to drug development are now conceptual, not technological.

What the future seems to hold is an array of synthetic drugs that will precisely alter brain function. If the pharmacologist is in charge of designing the product, the physician will in turn become an engineer who adapts the new technology to the needs and desire of the individual. Balancing molecules and case histories, the physician will mediate between new products and the idiosyncrasies of the patient.

Pharmacolgy and its psychiatric translations are about to emerge in a flurry of excitement and importance, analogous to those previously associated with particle physics and presently with the new genetics.

From the book; Intoxicating Minds by Ciaran Regan


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