RNA interference for human therapy

RNAi has emerged as a powerful technology for down-regulating gene expression, culminating in the Nobel prize award for Medicine in 2006. Research has shown that it could be effectively used in a variety of tissues to knock down target genes – especially those involved in disease – thus demonstrating the potential of RNAi in human therapy.

The EU 'RNA interference technology as human therapeutic tool' (RIGHT) initiative aimed to explore the technology of RNAi for therapy by delineating its underlying mechanisms. To achieve that, project partners had to overcome key technological barriers such as undesired interferon response and insufficient delivery, stability and targeting of RNAi inhibitors to the appropriate cells.

Selective reagents were generated for efficient delivery of chemically synthesised or vector-expressed siRNAs to cells and tissues of diseased organisms. Cell biology and disease models were used to assess the function and effectiveness of RNAi for the treatment of selected disorders. In particular, scientists showed the so-far unexplored potential of RNAi against influenza, arthritis, HBV and acute myeloid leukaemia (AML) in pre-clinical models.

Furthermore, the improved understanding of the molecular processes associated with RNAi and the naturally occurring microRNAs (miRNAs) is expected to have applications in healthcare, such as for the therapy of acute myeloid leukemia.

The RIGHT consortium demonstrated the efficacy of the RNAi technology as a therapeutic tool for many diseases. The knowledge generated will not only impact future research in the field but RNAi-based solutions are expected to get translated into clinical practice.

Provided by CORDIS