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Methodological Platform for Studies of Gene Functions from Cells to the Entire Organism

Coordinator: Prof. Dr. Wolfgang Wurst

Summary:



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RNA interference (RNAi) is one of the best tools available today for realising the full potential of the Human Genome Project by offering an efficient method of rapidly identifying, among the tens of thousands of genes, those which bear therapeutic potential. RNAi also offers the promise of a new, fast, cost-effective, and versatile complementation of current transgenic mouse knockout technologies.

The overall goal of the SMP RNAi is to further develop the RNAi technology with strong emphasis on the establishment of high resolution high throughput functional screens in tissue culture and to implement RNAi technology for functional assessment in vivo. These technologies promise to speed up functional annotation and validation of disease candidate genes and will therefore have a major impact on molecular dissection of disease mechanisms.

The SMP RNAi is structured as a pipeline to design, develop and implement RNAi technology in cell culture systems as well as for the study of embryonic and adult physiology in mouse models. In the overall structure of this SMP, two groups contribute to the different aspects of RNAi production and application in cell culture, two groups develop novel applications of RNAi during early midgestation embryogenesis, and two groups develop in vivo RNAi technology, and apply it to adult organisms, including phenotyping.

A strong bioinformatics group is included which supports design of experiments, evaluation of data and presentation to the scientific community. Cenix BioScience GmbH, a biotech company, is also part of the SMP. It has strong expertise in RNAi technology development and will offer RNAi services as well to NGFN-2 members as to the scientific community. Dissemination of knowledge will be performed by RiNA GmbH in form of workshops.

The SMP RNAi integrates all key technologies concerning RNAi design and construction currently available. In addition, it will further develop RNAi technology for large scale HT screening in human cells and for in vivo function studies in mice. It will bring together leading experts from the fields of RNAi development and application, cell biology, conditional gene mutagenesis and mouse genetics.

 
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