Objetivo
Sequence-specific alteration of genes in living organisms provides a new strategy for targeted gene repair or trait alteration that has in the last year seen a major breakthrough. The applying consortium has pioneered the toolkits for inducing site- specific sequence alterations at high rates in vivo, and has advanced the technology by using bioinformatics, biophysical, biochemical and molecular biology methods. By specifically stimulating homologous recombination through the creation of targeted site specific DNA double strand breaks, in vivo sequence alteration rates of several percent can be obtained. The double strand breaks are created either by site-specific designer zinc finger endonucleases or triple helix forming oligonucleotides. These advanced bio-molecules will be tested in an array of bio-essays using live cells to identify those with high activity and specificity, which later could be used in gene therapy protocols. Although the previously obtained results are above expectation, numerous improvements of the technology are necessary to increase specificity and efficacy, and to reduce side effects. We suggest to stepwise develop the technique further with the aim of applying this technology platform for the treatment of selected human genetic diseases.
Ámbito científico
- medical and health sciencesmedical biotechnologygenetic engineeringgene therapy
- natural sciencesbiological sciencesgeneticsDNA
- natural scienceschemical sciencesinorganic chemistrytransition metals
- natural sciencesbiological sciencesbiochemistrybiomolecules
- natural sciencesbiological sciencesmolecular biology
Convocatoria de propuestas
FP6-2005-LIFESCIHEALTH-6
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Régimen de financiación
STREP - Specific Targeted Research ProjectCoordinador
OSLO
Noruega