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RNA Interference Technology as Human Therapeutic Tool

Final Report Summary - RIGHT (RNA Interference Technology as Human Therapeutic Tool)

The RIGHT project aimed at exploiting the vast potential of RNA interference (RNAi) for human therapy. Based on the knowledge of gene sequences playing their role in diseases, RNAi molecules that specifically down-regulate the expression of dysfunctional genes can be generated, thus enabling a targeted therapy. RNAi has emerged as one of the most promising technologies in molecular and cell biology. The RNAi approach has been shown to work successfully in numerous tissues of therapeutic interest, indicating that RNAi may be useful for human therapy.

RIGHT combined the strengths of five synergistic competence domains to reach this ambitious goal and overcome key technological barriers such as undesired interferon response and insufficient delivery, stability and targeting of RNAi inhibitors to the appropriate cells. Rational and selective approaches have been taken to generate efficient RNAi reagents. Strategies have been developed for efficient delivery to cells and tissues of diseased organisms, using chemically synthesised siRNAs (small inhibiting RNA) and vector based gene therapy approaches using shRNA (small hairpin RNA). Cell biology and disease models have allowed addressing the function and effectiveness of RNAi for the treatment of selected diseases.

The RIGHT partnership of leading research institutions and biotech Small and medium-sized enterprises (SMEs) have delivered tools such as new enabling technologies and novel chemically synthesised and genetically generated inhibitors with efficient delivery properties. During these four and a half years, the potential of RNAi to diagnose and successfully treat severe unvanquished diseases have been demonstrated and proof of principle has been provided for the value of RNAi as a therapeutic tool in living organisms.

The results produced in RIGHT will impact both the health care industry, through the progress made in available diagnostic and therapeutic tools, and the research sector, by the provision of new technologies contributing to bring RNAi-based solutions from the labs to the patient.

The results from the assessment of optimised siRNAs in animal models constitute important new knowledge about the efficacy of RNAi as a therapeutic tool, which will have an impact on future research in the field. In particular, the demonstrated efficacy of the application of siRNA / shRNA / miRNA against different diseases such as influenza, arthritis, HBV and ACL in mice is a promising ground for further research about the potential therapeutic use of RNAi in these diseases. The improved understanding of the molecular processes associated with RNAi and the naturally occurring microRNAs (miRNAs) will also be used in future research about RNAi. Moreover, some of the patents filed further to research performed in RIGHT have an estimated application in healthcare within 5 - 10 years, such as the use of sisiRNA to improve the pharmacokinetic properties of siRNA in vivo and the use of miRNAs and siRNAs for the therapy of acute myeloid leukemia.