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Genome sequence breakthrough could hold key to new drugs

Genome sequence breakthrough could hold key to new drugs

Scientists in the US have potentially developed a new way to use genome sequence in the design of medicinal drugs.

Experts from the Florida campus of The Scripps Research Institute (TSRI) have released details of their discovery – using an example of a compound they have already established to tackle cancer cells. This potent amalgam effectively manipulates the poisonous cells, so they attack themselves and die.

It is hoped the way it was created can be adapted to lead to the formation of many other treatments.

The system involves gaining a better understanding into how drugs are bound to RNA folds, specifically microRNAs – which are tiny molecules apparent in almost all plant and animal cells. They regulate a range of cellular processes, working as "dimmer switches" for one gene or more by binding their transcripts and stopping them from being translated into proteins.

MicroRNAs are a relatively new discovery in the world of science, having only been identified for the first time in the 1990s. As a result, scientists are still learning about the potential they hold in regards to medical breakthroughs.

Using the example they have already developed, lead researcher Dr Matthew Disney said it was the first time therapeutic small molecules have been designed using RNA sequences alone, boasting it was "something many doubted could be done".

The study was part-funded by the National Institute of Health's National Institute of General Medical Sciences. Its spokesman Dr Peter Preusch commented: "This new work is another example of how Dr Disney is pioneering the use of small molecules to manipulate disease-causing RNAs, which have been underexplored as potential drug targets."

Further details of the research has now been published in the journal Nature Chemical Biology. Dr Disney has described his team's findings so far as "unprecedented" and something that "provides great excitement for future developments".