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Subproject structure and research activities

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1. Molecular Mechanisms and Technologies
Novel molecular strategies for application of RNAi as a therapeutic tool are developed based on an improved understanding of the molecular processes associated with RNAi and the naturally occurring counterpart microRNA. Use is made of large-scale libraries and high-throughput screening technologies.
Right-schema 2. Chemical Tools
Improved inhibitors are synthesized chemically and extensively tested in cell culture and living organisms in order to increase sensitivity, specificity and cost-effectiveness and reduce side effects. New chemical tools will include: RNA mimics, RNA conjugates and delivery reagents.
3. Genetic Tools
Potent viral or non-viral delivery RNAi vectors are generated and their features evaluated in relation to their chemical counterparts. Special emphasis is given to the development of tissue-specific and inducible systems.
4. Pharmacokinetics
For the development of a drug, synthetic or genetic RNAi reagents are first assessed in cultured cells with pharmacokinetic methods. Successful candidates are assessed in animal models, and extensive phenotyping of treated animals will be performed
5. Cell Biology and Disease models
Selected disease models are used for the paradigmatic assessment of RNAi as a therapeutic tool to generate RNAi leads for clinical tests. In particular RIGHT is focussing on infectious diseases and genetic defects causing degenerative diseases and cancer.

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