Non-invasive administration of adeno-associated viral vectors

Details Non-invasive administration of adeno-associated viral vectors Non-invasive administration of adeno-associated viral vectors

C - Chemistry, Metallurgy

12

N

C12N 15/86 (2006.01) A61K 9/08 (2006.01) A61K 31/70 (2006.01) A61K 38/18 (2006.01) A61K 38/21 (2006.01) A61K 38/48 (2006.01) A61K 48/00 (2006.01) C12N 15/864 (2006.01)

Patent

CA 2266619

The present invention provides new methods of administering recombinant AAV vectors for effective, long-term expression of a gene of interest in animals, including humans. Provided herein are methods of subcutaneously injecting recombinant AAV vectors containing a gene of interest encoding any diffusible polypeptide, ribozyme, nucleic acid, or antisense oligonucleotide for gene therapy. The methods of this invention provide significant advantages, including the ability to deliver any diffusible polypeptide or nucleic acid of a defined size via subcutaneous injection and obtain high-level, long-term expression in regions approximate or distant from the site of subcutaneous injection.

Nouveaux procédés pour administrer des vecteurs recombinés de virus AAV pour produire une expression efficace, longue durée, d'un gène d'intérêt chez des animaux, y compris chez l'homme. L'invention concerne des méthodes d'injection sous-cutanée de vecteurs AAV recombinés contenant un gène particulier codant n'importe quel polypeptide, ribozyme, acid nucléique, ou oligonucléotide antisens diffusibles en vue d'une thérapie génique. Ces méthodes offrent des avantages significatifs, parmi lesquels la possibilité d'administrer n'importe quel polypeptide ou acide nucléique diffusible de dimension prédéterminée par injection sous-cutanée, pour obtenir une expression importante et longue durée, dans des régions proches ou distantes du site d'injection sous-cutanée.

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