Thermo Fisher claims breakthrough with gene-silencing agent

By Wai Lang Chu

- Last updated on GMT

Related tags: Rna

Research into the use of RNA interference (RNAi) to "silence" gene
activity has taken another step forwards, saysThermo Fisher
Scientific which has launched an agent that accelerates the
processes in which RNAi is used to shut off specific genes.

The breakthrough technology laughs in the face of conventional methods, which currently find the process of getting RNAi inhibitory agents inside cells very difficult. The agent could well have applications in drug discovery and research in many life sciences fields such as neuroscience, immunology, stem-cell science and oncology. What's so special about the Thermo Scientific's Dharmacon Accell short-interfering RNA (siRNA) is its ability to be absorbed directly by cells without the use of conventional delivery methods such as transfection reagents, viruses or electroporation. Dharmacon Accell siRNA is mixed with the optimised Accell delivery media and then added to cultured cells. Thermo claim its two-step process saves researchers significant time in laboratories and avoids the experimental variability caused by toxicity and off-target effects of conventional siRNA delivery methods. "Dharmacon Accell is the only siRNA molecule that has been able to penetrate every cell type we have tested, without the need for delivery reagents that can skew experimental results​," said Dr Ian Jardine, vice president of global research and development for Thermo Fisher Scientific. Thermo said that preliminary studies also suggested that long-term silencing of up to 30 days could be achieved through repeated application of Dharmacon Accell siRNA - a feat previously not possible with conventional synthetic siRNA reagents. If this turns out to be the case, Thermo Fisher may have found a way to extend the crucial window of conditions in which conventional siRNA delivery is effective. In addition, the elimination of delivery reagents, along with the associated non-specific effects, greatly simplifies the interpretation of RNAi experiments. RNAi is a powerful technology used to silence, or knock down, target genes and the proteins they produce, revealing their role in biological pathways and disease processes. Understanding gene function is critical for the development of drugs targeting those genes. "Previous technologies for delivering siRNA into difficult cell types, such as primary cells, suspension cells, stem cells and neurons, were often ineffective and resulted in high levels of cell death​," added Jardine. "With Dharmacon Accell siRNA and our Dharmacon SMARTvector shRNA advancement, scientists now have an effective RNAi tool set for biomedical and pharmaceutical research that will have broad implications for human health​." RNAi has proved its value ever since the technique was discovered in 2006 by two U.S biologists, who had discovered a way to shut off individual genes by blocking key RNA molecules in the cell. But while the technique immediately became a powerful research tool, it was unclear how quickly it could be translated into drugs to treat the myriad health conditions that crop up when gene activity is out of whack. Thermo Fisher are not the only outfit investigating the use of RNA interference, which has managed to pick up a head of steam lately. Only a couple of weeks ago Alnylam Pharmaceuticals, reported on data illustrating the effectiveness of an RNAi-based treatment in humans. Its Phase II Clinical Trial of ALN-RSV01 demonstrated statistically significant anti-viral efficacy with an approximately 40 per cent reduction in RSV infection rate and 95 per cent increase in infection-free subjects. "This data demonstrates significant anti-viral activity for an RNAi therapeutic in a major respiratory disease, an accomplishment that is notable in clinical medicine​," said John DeVincenzo, Principal Investigator for the study known as GEMINI. "A positive outcome for ALN-RSV01 represents the first new approach in decades for a drug demonstrated to have human anti-viral efficacy against RSV. These data support continued development of the RNAi therapeutic for the treatment of RSV infection, a major unmet medical need​," he added.

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