Gene silencing may be future of medicine

Two American scientists won the Nobel Prize for medicine last month for their groundbreaking discovery of how to switch off genes.

Not only were these scientists on the young end of the scale for winning such a major prize, but it was very quick recognition for their work, which was only published eight years ago. This recognition came because the work of Andrew Fire and Craig Mello has the potential to be a critical step in the quest for new treatments for all kinds of diseases.

This research showed that a double strand of ribonucleic acid (RNA), which operates as the genetic messenger in cells, can silence or turn off specific genes in a process called RNA interference (RNAi).

Since this discovery in 1998, the idea of RNAi has grown into a popular area of research as companies and academic institutions see its potential for targeting a myriad of conditions that involve our genetic makeup, or others such as viruses that may have a genetic code of their own that could be altered or made inert through RNAi.

Turning off specific genes can help to teach us exactly what those genes do in a particular disease, knowledge which will prove extremely valuable to the future of medicine.

Right now, RNAi is being used extensively in research labs around the world. Clinical trials for a variety of viral diseases, eye disorders and cardiovascular diseases are underway and this method is also being used to better understand and kill HIV. Further, this process may lead to ways to stop gene expression in diseases such as cancer by slowing tumour growth.

In psychiatry, RNAi may have promise for any number of conditions since it is believed that most have a genetic component.

For most such conditions we do not yet know exactly which genes are involved or what those genes do. There are candidate genes, ones that we think are likely involved, but our knowledge of their function is quite rudimentary.

Since the human genome is only slightly different from that of many other animals (1.23% different base pairs between human and chimp genomes), it may be possible to identify the candidate gene counterparts in other animals and by turning them on and off figure out what they do.

Eventually, of course, if we find out that a disorder results from the overactivity of a particular gene we would then have a means of turning it off. This could be potentially curative for some disorders.

Eventually by means of comparsions between organisms and the ability to turn specific genes on or off we should be able to identify exactly what each gene does and why one organism is different from another.

Microsoft billionaire Paul Allen has funded the Allen Institute for Brain Science which has just completed a digitized index of the more than 21,000 genes that are active in the mouse brain and has posted these results online, available to researchers at no cost. The same is now being done for the human neocortex. All of this will help move us forward in understanding the role that specific genes may play in various conditions and may be groundbreaking in future treatments.

RNAi is just one more area where continued extensive research is needed in order to truly discover its potential in medicine. It may well be a technology that will be at the forefront of treating and curing disease in the future, giving all of us longer, healthier lives and saving the healthcare system billions of dollars on treatments that only slow or manage serious conditions rather than eliminating them.


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