The Strange Case of Molecular Medicine

Abstract

“Modern medicine is entering the “molecular age” — a statement similar to this is frequently found not only in the writings of physicians and scientists, but also in the mass media. Like many of the paradigm shifts that have characterized technical progress in the medical arena, the characterization of diseases at the level of genes and molecules is only the latest step in the search for the final frontier of medicine. Over the past centuries, medical research has continuously refined its level of organization, as it has focussed on the whole body, then on the levels of the organ, the cell, and now, finally, on molecules and genes. The term “molecular medicine” describes the impact of molecular biology and genetics on medicine and characterizes a new field devoted to the search for molecular aspects in disease etiology, but also to the potential use of the resulting new technology for molecular diagnosis and therapy. The human genome project has now been completed, generating an abundance of new genetic information that can be applied to medicine. New candidate genes for human disease will be defined and will be tested in functional models. This information can be used in many ways: DNA technology has been and will be useful to define genetic risk factors for diseases long before they appear, opening the door for preventive strategies. Furthermore, this genetic information can be used to optimize conventional pharmacological treatment, improving the rate of responders to drug treatment which is currently low in many situations. This means that the individual gene profile can be used to choose the best treatment option for the patient. Lastly, gene therapeutic approaches have been proposed using somatic gene transfer technology. These techniques are targeting the repair of defective genes, altering gene regulation by increasing “good” genes and inhibiting “bad” genes. Alternatively, functional genes can be applied to replace defective genes or genes thought to be protective for defined pathophysiological conditions can be added to the organism. All these approaches offer many new and fascinating ways to allow for tremendous progress in medicine.

“ I was led to remark that, whereas, in the beginning, the difficulty had been to throw off the body of Jekyll, it had of late gradually but decidedly transferred itself to the other side. All things therefore seemed to point to this: that I was slowly losing hold of my original and better self, and becoming slowly incorporated with my second and worse.”

From “The strange case of Dr. Jekyll and Mr. Hyde” by Robert Louis Stevenson. 1886