'Gene Targeting' Innovators Receive Nobel Prize
This year's Nobel Prize for medicine has been awarded to a trio responsible for significant innovation in the field of gene technology. The group is comprised of a pair of US scientists - Mario Capecchi and Oliver Smithies - and Martin Evans - their British collaborator. Together, they devised the technique of 'gene targeting', which provided them with the ability to simulate human diseases in rodents through introducing generic variations into their stem cells. In support of their decision, the Nobel Committee highlighted how this technique had opened up many new avenues in respect of conditions including cancer and cardiovascular disease.
For example, the medical research industry now has a better comprehension of how disease can afflict people previously considered healthy.
Gene targeting has also assisted in providing insight on the process of ageing, and new information on how humans develop pre-birth. On the wider scale, it can be employed in the study of virtually any angle relating to physiology among mammals.
The Nobel Committee lauded gene targeting as "an immensely powerful technology", which nearly every strand of research into biomedical issues had now adopted. "Gene targeting in mice has pervaded all fields of biomedicine," said the committee, adding: "Its impact on the understanding of gene function and its benefits to mankind will continue to increase over many years to come."
Gene targeting is known by an alternative name: gene 'knockout'. It enables scientific researchers to isolate specific genes so, ultimately, the spread of a disease can be assessed on a gene-by-gene basis. So far, in excess of 10,000 mice genes have undergone the process.
Consequently, over 500 different replicas of human diseases have been created.
The three scientists involved have gone on to employ gene targeting/knockout in further research. Professor Capecchi has used it to reveal the part genes play in the development of human organs, while Sir Martin has focused primarily on the disease cystic fibrosis. Finally, Professor Smithies' production of so-called "mouse models" has placed more focus on blood-related diseases.
The University of Cambridge's Professor Stephen O'Rahilly highlighted how "The development of gene targeting technology in the mouse has had a profound influence on medical research."
He continued: "Thanks to this technology we have a much better understanding of the function of specific genes in pathways in the whole organism and a greater ability to predict whether drugs acting on those pathways are likely to have beneficial effects in disease."
Source - Pharmaceutical International's Research and Development Analyst
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