The RIGHT project aims at exploiting the vast potential of RNA interference (RNAi) for human therapy, based on an advanced understanding of the underlying mechanisms. Rational and selective approaches will be taken to generate efficient RNAi reagents, and strategies will be developed for efficient delivery to cells and tissues of diseased organisms. RIGHT combines the strengths of 5 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 to the appropriate cells. 1. The understanding of the molecular processes associated with RNAi and microRNA will be improved as a basis for the development of molecular strategies and tools enabling the successful application of RNAi for human therapy. 2. Improved inhibitors including RNAi mimics and potent delivery reagents will be chemically synthesized and extensively tested in cell culture and living organisms in order to increase sensitivity, specificity and cost-effectiveness and reduce side effects. 3. Potent viral or non-viral RNAi vectors will be generated and their features evaluated in relation to their chemical counterparts. 4. For the development of a drug, synthetic or genetic RNAi reagents will be assessed with pharmacokinetic methods, and extensive phenotyping of treated animals will be performed. 5. Selected disease models will be used for the paradigmatic assessment of RNAi as a therapeutic tool to generate RNAi leads for clinical tests. The RIGHT partnership of leading research institutions and biotech SMEs will deliver tools such as new enabling technologies, chemically synthesized and genetically generated inhibitors with efficient delivery properties. Within 4 years, the potential of RNAi to diagnose and successfully treat severe unvanquished diseases will be demonstrated and proof of principle provided for the value of RNAi as a therapeutic tool in living organisms.
Keywords:
RNA interference, siRNA, shRNA, human therapy, microRNA, RNAi, RNAi vectors, chemistry, cell biology, clinical research, gene therapy, viral and non-viral vectors, diagnostics, untranslated RNA
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