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Contenido archivado el 2022-12-23

Development of synthetic gene transfer vectors for gene therapy

Objetivo



Participants in the project will develop efficient approaches for delivery of nucleic acids and oligonucleotides into cells.

The Russian group will investigate kinetics and mechanisms of uptake of nucleic acids, oligonucleotides and oligonucleotide analogues by various cells and identify proteins involved in internalisation of nucleic acids. The group will synthesise new constructions possessing cholesterol and polyamine groups, for improving cellular uptake of polynucleotides. The group will study pharmacokinetics of oligonucleotides and polynucleotides, and the effect of lipopolyamines and other vectors on the fate of nucleic acids in the organism.

The French group will design and test lipopolyamine constructions and compositions as oligonucleotide- and gene transfer vectors. The group will find optimal composition of the vector constituents and will elaborate optimal physico-chemical procedures for the formation of virus-like particles composed of nucleic acids and vector molecules.

The Italian group will carry out the final testing of the developed techniques on animal models and on human patients.

The results of these studies will provide knowledge needed for optimal use of natural cellular uptake mechanisms for efficient delivery of oligonucleotides and polynucleotides into cells in culture and in vivo. Another expected result is the development of efficient artificial vectors for gene transfer techniques which will find application in research, in gene therapy, and for enhancement of efficiency of oligonucleotide derivatives in vivo, which may considerably improve the therapeutic potential of oligonucleotide-based antiviral and anticancer therapeutics.

Convocatoria de propuestas

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Régimen de financiación

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Coordinador

Université Louis Pasteur Strasbourg
Aportación de la UE
Sin datos
Dirección
Route du Rhin 74
67401 Illkirch
Francia

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Coste total
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Participantes (2)