Novel liposomal vector complexes and their use in gene therapy

Details Novel liposomal vector complexes and their use in gene therapy Novel liposomal vector complexes and their use in gene therapy

C - Chemistry, Metallurgy

12

N

C12N 15/88 (2006.01) A61K 9/127 (2006.01) A61K 47/48 (2006.01) A61K 48/00 (2006.01) A61P 35/00 (2006.01) C12N 15/87 (2006.01)

Patent

CA 2375854

The invention relates to a novel liposomal vector complex, containing the following components: a) a nucleic acid sequence of any length; b) a cationic support which condenses the component a) and is lysosomolytic and/or lysosomotropic; c) lipids and phospholipids which form a liposome; d) optionally a ligand which has a connecting point for a target cell; and e) optionally a fusogenic substance which can replace the lysosomolytic and/or lysosomotropic functions of component b). If a fusogenic substance (e) is present, the cationic support (b) need not be lysosomolytic and/or lysosomotropic. The invention also relates to the production and use of said complex.

La présente invention concerne un nouveau complexe vecteur liposomal comprenant les composants suivants : a) une séquence d'acide nucléique de longueur indifférente; b) un support cationique qui condense le composant a) et est lysosomolytique et/ou lysosomotrope; c) des lipides et phospholipides formant un liposome; d) éventuellement un ligand possédant un site de liaison pour une cellule cible; et e) éventuellement une substance fusogène qui peut remplacer la fonction lysosomolytique et/ou lysosomotrope du composant b). En présence d'une substance fusogène (e), le support cationique (b) ne doit pas être lysosomolytique et/ou lysosomotrope. Cette invention concerne également la production de ce complexe et son utilisation.

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