Cell-specific gene delivery vehicles

Details Cell-specific gene delivery vehicles Cell-specific gene delivery vehicles

A - Human Necessities

61

K

A61K 47/48 (2006.01) A61K 31/70 (2006.01) A61K 38/45 (2006.01) A61K 48/00 (2006.01) A61K 49/00 (2006.01) A61K 49/08 (2006.01) A61K 51/06 (2006.01)

Patent

CA 2202478

A delivery vehicle is described that is capable of being specifically bound to and taken into targeted cells, delivering numerous physiological agents, particularly paramagnetic ions for magnetic resonance imaging (MRI) of the cells. The delivery vehicle comprises a polymeric molecule having a net positive charge complexed with another polymeric molecule having a net negative charge. Cell targeting moieties and physiological agents, including contrast agents and therapeutic agents, are attached to one or both of the polymeric molecules. In one embodiment, the polymeric molecule having a net negative charge is a nucleic acid. Thus, the delivery vehicles can be used in clinical protocols in which nucleic acids for gene therapy and agents for MRI contrast are co-transported to specific cells allowing medical imaging monitoring of nucleic acid delivery.

La présente invention concerne un véhicule de transfert capable de se lier spécifiquement à des cellules ciblées et d'y pénétrer, transférant ainsi de nombreux agents physiologiques, en l'occurence des ions paramagnétiques destinés à l'imagerie par résonance magnétique (IRM) des cellules. Ce véhicule de transfert comprend une molécule polymère de charge nette positive formant un complexe avec une autre molécule polymère de charge nette négative. Des fractions de ciblage cellulaire et des agents physiologiques, y compris des agents de contraste et des agents thérapeutiques, sont fixés à une seule molécule ou au deux molécules. Selon un mode de réalisation, la molécule polymère à charge nette négative est un acide nucléique. De tels véhicules de transfert conviennent aux protocoles cliniques nécessitant, pour la surveillance par imagerie médicale de l'administration de l'acide nucléique, le transport combiné des acides nucléiques de thérapie génique et des agents de contraste d'IRM vers des cellules spécifiques.

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