Composition For Delivery Of Genetic Material

Abstract

The present invention relates to exosomes, loaded with genetic material and methods of producing them and to the use of such exosomes for delivering genetic material in vivo, in particular the use of such exosomes in methods of gene therapy or gene silencing.

1 . A composition comprising an exosome, wherein the exosome is loaded with exogenous genetic material. 
     
     
         2 . A composition according to  claim 1  wherein the exosome is derived from dendritic cells. 
     
     
         3 . A composition according to  claim 1  wherein the exogenous genetic material is a DNA plasmid encoding a therapeutic protein or an immunogen. 
     
     
         4 . A composition according to  claim 1  wherein the exogenous genetic material is an siRNA. 
     
     
         5 . A composition according to  claim 3 , wherein the plasmid encodes a therapeutic protein for use in a method of gene therapy. 
     
     
         6 . A composition according to  claim 3  wherein the plasmid encodes an immunogen, for use in a method of generating an immune response to the immunogen. 
     
     
         7 . A composition according  claim 1 , wherein said exosome comprises a targeting moiety expressed on the surface of the exosome. 
     
     
         8 . A composition according to  claim 7  wherein the targeting moiety comprises a peptide which binds to a moiety present on the cell to be targeted. 
     
     
         9 . A composition according to  claim 8  wherein the exosome comprises an exosomal transmembrane protein which has been modified to incorporate the peptide targeting moiety. 
     
     
         10 . A composition according to  claim 9  wherein the exosomal transmembrane protein is selected from Lamp-1, Lamp-2, CD13, CD86, Flotillin, Syntaxin-3. 
     
     
         11 . A composition according to  claim 4  wherein the exosomal transmembrane protein is Lamp-2b. 
     
     
         12 . A composition comprising an exosome containing genetic material, wherein the exosome is derived from an immature dendritic cell, for use in a method of delivering the genetic material in vivo. 
     
     
         13 . A method of gene silencing in a non human animal model, comprising administering to the animal an exosome according to  claim 4 , wherein the siRNA is directed against the gene to be silenced. 
     
     
         14 . A method of loading exosomes with genetic material comprising providing a composition of exosomes, and loading the exosomes with genetic material by electroporation. 
     
     
         15 . A method according to  claim 14 , wherein the electroporation is carried out at a voltage between 20 v/cm to 1 OOv/cm. 
     
     
         16 . A method of loading exosomes with genetic material comprising providing a composition of exosomes and loading the exosomes with nucleic acid by transfection, using a cationic liposome transfection agent. 
     
     
         17 . A method according  claim 14 , wherein the exosomes are derived from dendritic cells. 
     
     
         18 . The method according to  claim 16 , wherein the exosomes are derived from dendritic cells.