Objectif
Sequence-specific alteration of genes in living organisms using small oligomers, as has been recently successfully demonstrated by us, could provide a new strategy for targeted gene repair or trait alteration. The applying consortium has in the last years developed a concept of using small dual oligomers to introduce in vivo site-targeted specific sequence alterations, and has advanced the toolkit for synthesis and functional testing of these oligomers using biophysical, biochemical and molecular biology methods.
The oligomers (ROs) are composed of two functional domains: a targeting domain that provides high sequence affinity to any desired location in the genome, and a template domain that activates the cellular repair machinery by active group-transfer to induce small sequence changes without leaving foreign sequences. Hitherto the consortium has reached its goal of the 5th framework defined as in vivo rates of targeted sequence correction over 2% on a standardised cellular readout.
The goal of the suggest ed work in the 6th framework is to further improve the design of platform technology to induce precise single-base changes in a cell culture readout at rates between 2 and 5% and in a selected disease locus without collateral damage. ROs provide a new paradigm for in vivo genetic engineering and a novel tool to be tested for its use in somatic gene therapy.
Champ scientifique
Mots‑clés
Thème(s)
Appel à propositions
FP6-2003-LIFESCIHEALTH-I
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Régime de financement
STREP - Specific Targeted Research ProjectCoordinateur
OSLO
Norvège