Systemic viral/ligand gene delivery system and gene therapy

C - Chemistry – Metallurgy – 12 – N

Patent

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Details

C12N 15/86 (2006.01) A61K 38/17 (2006.01) A61K 47/48 (2006.01) A61K 48/00 (2006.01) C12N 15/87 (2006.01)

Patent

CA 2349238

The present invention relates to gene transfer and gene therapy technology. More specifically, the invention provides compositions and methods for targeted virus delivery. The method utilizes a method of mixing the virus, which may be a recombinant virus which will express a protein of interest or a nucleic acid of interest, with a cell-targeting ligand, e.g., transferrin. The virus and ligand are mixed without crosslinkers or agents which would covalently bond the virus and ligand. This simple mixing causes less inactivation than chemically linking the ligand to the virus and therefore results in a more active therapeutic composition than obtained by methods which utilize crosslinking agents.

La présente invention concerne le transfert génique et la technologie de thérapie génique, notamment des compositions et des procédés d'apport ciblé de virus. Le procédé se fonde sur l'utilisation d'un procédé qui mélange des virus, qui peuvent comprendre un virus recombinant exprimant une protéine ou un acide nucléique d'intérêt, et un ligand tel que la transférine ciblant une cellule. On mélange le virus et le ligand sans utiliser d'agents de réticulation ou d'agents liant par liaison covalente le virus et le ligand. Ce mélange simple provoque une plus faible inactivation en comparaison à la liaison chimique du ligand au virus et permet de ce fait de créer une composition thérapeutique plus active que celles obtenues par des procédés recourant à des agents de réticulation.

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