Composition for delivery of genetic material
View Patent ↗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 method of targeting an exosome to a selected tissue or cell type, comprising expressing a targeting moiety as a fusion protein between the targeting moiety and an exosomal transmembrane protein on the surface of the exosome, wherein the targeting moiety is a peptide of from 5 to 100 amino acids in length, and binds a cell surface moiety expressed on the surface of the selected tissue or cell type, wherein the exosomal transmembrane protein is selected from Lamp-1, Lamp-2, CD13, Flotillin, Syntaxin-3, CD44, ICAM-1, Integrin alpha4, L1CAM, LFA-1, Vti-1A and B, CD9, CD37, CD53, CD63, CD81, CD82 and tetraspanins.
2. The method according to claim 1 , wherein the targeting moiety targets the exosome to tissue or cells selected from muscle, brain, liver pancreas and/or lung.
3. The method according to claim 1 , wherein the targeting moiety targets the exosome to diseased tissue or diseased cells.
4. The method according to claim 1 , wherein the targeting moiety is separated from the transmembrane protein by a spacer or linker sequence.
5. The method according to claim 4 , wherein the spacer or linker sequence is a peptide of from 1 to 5 amino acids.
6. The method according to claim 5 , wherein the spacer or linker comprises alanine, arginine, serine and/or glycine residues.
7. The method according to claim 1 , wherein the exosome is loaded with exogenous genetic material.
8. The method according to claim 3 , wherein the diseased tissue is a tumour tissue, or the diseased cell is a tumour cell.