Down regulation of the gene expression by means of nucleic acid-loaded virus-like particles
OwnerJens Gruber, Wolfgang Lükeinventors
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Abstract
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.
Claims32
A composition, comprising a virus-like particle (VLP) wherein said VLP comprises at least one viral capsid protein, and wherein at least one RNA-interference (RNAi-) inducing molecule is included in the interior of the VLP, wherein said VLP comprises at least one viral capsid protein that is modified with a heterobifunctional linker molecule that allows for a retargeting of said VLP, wherein said VLP comprises the capsid protein VP1 of a human polyoma JC virus, and wherein the heterobifunctional linker molecule is bound to at least one lysine residue on said capsid protein VP1 of the human polyoma JC virus, wherein the lysine residue corresponds to K60 or K164 of SEQ ID NO:2.
Dependencies are inferred from the claim text.