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RNA interference – a new gene therapy tool

The unexpected discovery of RNA interference resulted from experiments on petunias. The time is long gone when RNA molecules were regarded as the mere servants of DNA. Nowadays, RNA molecules are seen as one of the key biochemical substances in the metabolism of living organisms. In addition, the discovery of RNA interference (RNAi) has given researchers a promising tool. There has been enormous progress. Hardly five years have passed since the discovery of RNAi. Today, research focuses on the potential therapeutic benefits of this naturally occurring mechanism.

The unexpected discovery of RNA interference resulted from experiments on petunias. (Photo: Wikipedia)
The unexpected discovery of RNA interference resulted from experiments on petunias. (Photo: Wikipedia) 
A lecture praising the discoverers of RNAi, Craig Mello and Andrew Fire, who were awarded the Paul Ehrlich Ludwig Darmstaedter Prize in 2006, drew attention to “RNA interference as a comparatively simple and universal method used for gene silencing, in which messenger RNA is degraded in a complex mechanism involving small, double-stranded RNA molecules, thereby reducing the expression of proteins involved in pathological processes. RNAi has already made an invaluable contribution to our understanding of the molecular and medically relevant relationships” (source: Ärztezeitung, 13.3.2006). Gene silencing is important for the regulation of genes and protects the cells from pathogens such as RNA viruses.

Popularity of the simple and universal molecule

Since then, scientists have agreed that RNA (siRNA, miRNA, small RNA, …) is the regulatory system of cells whose activation or disturbance can cause diseases. Many biomedical experts have great hopes for RNAi as a therapeutic tool in so far as a growing number of companies are dreaming of achieving high ROIs. It has been shown that RNA fragments (21 to 25 nucleotides long) can be introduced into human cells in order to suppress protein synthesis. Scientists have already succeeded in suppressing the proliferation of HIV in the test tube.

Truffle pigs for big pharma?



The pharmaceutical industry is increasingly interested in RNAi technology because genes are important targets for the development of drugs. The number of cooperative agreements between big pharmaceutical companies and RNA companies is growing, as shown by Internet portals such as www.rnai.net. There are approximately 5000 disease-associated genes.

RNAi-based drugs have the potential to be more selective and hence more effective and less toxic than traditional drugs. Some people even envisage the creation of a new class of drugs – on the condition that further progress is made in RNAi therapy (Pharmacogenomics 6/Dec 2005). Three US American companies are testing the effect of RNAi fragments in clinical trials investigating whether these fragments might be able to stop the proliferation of blood vessels in the eyes (macular degeneration). Sceptics hold that this disease is a specific case and cannot be transferred to other diseases.

Initial drawbacks for RNAi therapies

However, there were initial drawbacks. Not so long ago, a US researcher reported (Nature 441/p. 537-541; 25.5.2006) that a number of mice suffering from hepatitis C died following RNAi therapy. Nevertheless, experts still regard it as likely that the first RNAi drugs will receive marketing authorisation in 2010. Further efforts are being made to test RNAi for other diseases in clinical trials (e.g. SR Pharma plc's subsidairy Atugen). But care must be taken: gene therapy also started off at a furious rate.
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http://www.bio-pro.de/magazin/thema/00163/index.html?lang=en