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Study of RNA Silencing to Treat Life-Threatening Diseases

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Scientists from The Sainsbury Laboratory are leading a new research consortium to study how RNA silencing could be used to treat life-threatening diseases. The European Commission has committed 11.8 million euros to this four-year Integrated Project funded under the Sixth Framework Programme.

“RNA silencing, also called RNA interference, is the cell’s natural ability to turn off genes”, said Professor David Baulcombe of the Sainsbury Laboratory at the John Innes Centre.

“Only a few years ago it was unknown, but now RNA silencing is one of the most powerful tools available to researchers. We can use it to understand the function of genes and the mechanisms of cellular regulation. We can also use it as a diagnostic tool for cancer and other diseases. In future it may also be possible to use RNA silencing as the basis of novel therapy for diverse diseases ranging from avian influenza to cancer,” Baulcombe continued.

RNA silencing is thought to have evolved as a defence mechanism against viruses. In primitive cells it was a type of immune system that could recognize and then silence viral genes. Later in evolution the silencing mechanism was recruited for switching off genes involved in normal growth of cells and responses to stress.

It occurs in all sorts of organisms from yeasts to humans and the recent discoveries have revealed a previously unknown role for RNA (ribonucleic acid). They have shown how, in addition to the previously understood role as a cellular messenger that directs protein synthesis, RNA can also silence expression of genes. By introducing specific silencing RNAs into an organism, the expression of genes can be turned down in a controlled way.

“Although there has been rapid recent progress in understanding RNA silencing there is still much to be done” said Professor Baulcombe.

“For example we need to ensure that an RNA targeted against gene X will only silence gene X and nothing else. When we can do that we will be able to use RNA as a drug without side effects. We also need to understand more about the role of silencing RNAs in normal growth and development. That information will then allow us to use the presence of silencing RNAs to diagnose disease states in a cell,” he added.

Stimulated by the great potential of RNA silencing the European Commission has funded a consortium of the leading European laboratories. The consortium includes researchers working on RNA silencing in model plant and animal systems as well as humans.

The use of the model systems allows experiments to be carried out that would be impossible with humans although the new discoveries may be translatable into new technologies for use in medicine.

Seventeen world-class laboratories and companies from nine European countries are members of the SIROCCO consortium. SIROCCO stands for “Silencing RNAs: organisers and coordinators of complexity in eurkaryotic organisms”.