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Ex vivo gene delivery for stem cells of clinical interests using synthetic processes of cellular and nuclear import and targeted chromosomal integration


Our aim is to develop SyntheGeneTransfer (SGT), a new ex-vivo gene delivery protocol to provide stable long-term expression of integrated transgenes. Our clinical objective is to provide gene therapy solutions for some genetic diseases of the neuromuscul ar and skeletal systems as well as circulating-polypeptide deficiencies that together afflict over 35,000,000 patients in Europe. We have therefore selected mesenchymal and muscle stem cells as model systems in which to develop the SGT protocol.The SGT p rotocol is designed to overcome three key barriers: transgene internalization in stem cells, transport within the nucleus and integration at a predefined "safe" genomic locus that will permit transgene expression. To achieve these goals and fully develop the technology, this application draws together a multi-disciplinary European team from four public laboratories and two SMEs. To overcome serious problems with current transfection methods, the teams will develop protocols for internalization and nucle ar membrane transport based on Lipoplex, block copolymers and Bioplex. To overcome serious problems with current integration systems, a new targeted transposition system will be developed using a Mos1 transposon.Our main technical objective is to enginee r SGT to provide a method for the integration of large transgenes that will be highly efficient and maintain a stringent safety by preventing the oncogenic transformation of the target cells. Optimization of the cellular internalization and nuclear impor t steps will allow the cells to be treated with a minimal amount of the transgene. This will minimize the probability of illegitimate integration events at unexpected genomic loci. To optimize the integration step of the process, the Mos1 transposon will be engineered to allow targeting to specific loci.The system will also be engineered to accept large transgenes without the concomitant loss of efficiency of observed with existing systems.

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