Down-regulation of the gene expression by means of nucleic...

C - Chemistry – Metallurgy – 12 – N

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C12N 15/11 (2006.01) A61K 31/713 (2006.01) A61K 47/42 (2006.01) C12N 7/04 (2006.01)

Patent

CA 2699638

The present invention relates to compositions of virus-like particles for the introduction of RNA-interference (RNAi-) inducing molecules into eukaryotic cells and methods for the cell type-specific transduction of a plurality of eukaryotic cells with RNAi- inducing molecules. The present invention furthermore relates to methods for a diagnosis, prevention and/or treatment of diseases or disease states associated with an increased expression rate of at least one endogenous gene, and/or with the undesired expression of at least one endogenous gene and/or foreign nucleic acids, in particular viral nucleic acids.

La présente invention concerne des compositions de particules pseudovirales, permettant d'incorporer dans des cellules eucaryotes des molécules induisant l'ARN interférence (ARNi), ainsi que des procédés permettant de réaliser une transduction spécifique du type de cellule d'une pluralité de cellules eucaryotes contenant des molécules induisant l'ARNi. L'invention concerne également des procédés de diagnostic, de prévention et/ou de traitement de maladies ou d'états pathologiques, qui s'accompagnent d'un taux d'expression accru d'au moins un gène endogène et/ou de l'expression indésirable d'au moins un gène endogène et/ou d'acides nucléiques étrangers, notamment d'acides nucléiques viraux.

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