Down regulation of the gene expression by means of nucleic acid-loaded virus-like particles
OwnerJens Gruber, Wolfgang Luekeinventors
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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.
Claims21
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.
A method for introducing RNAi-inducing molecules into a target cell, comprising the steps:
- (i) assembly of the viral capsid proteins into a VLP in the presence of the RNAi-inducing molecules,
- (i) contacting the VLP loaded with the RNAi-inducing molecules with the target cell under conditions that allow for an uptake of the RNAi-inducing molecules into the target cell.
A method for the therapy and/or diagnosis of a disease or a disease state are caused by the expression of a nucleic acid that stems or is derived from a pathogenic organism, or by the increased or undesired expression of an endogenous gene, wherein said method comprises administering, to a subject in need of such therapy and/or diagnosis, a composition of claim 39.
A modified JCV-VP1 capsid protein comprising a heterologous nuclear localization signal, a nucleic acid encoding therefore;
- or a VLP-composition containing said modified JCV-VPI capsid protein.
A VLP-composition, wherein at least one immune stimulatory nucleic acid, optionally in combination with polypeptide or peptide immunogen, an aptamer or an siDNA-molecule, is included in the interior of the VLP.
A JCV-VP1 capsid protein to which at least one heterologous binding partner is bound via the amino acid residues K60 and/or K164.
Dependencies are inferred from the claim text.