Serpin enzyme complex receptor-mediated gene transfer

Details Serpin enzyme complex receptor-mediated gene transfer Serpin enzyme complex receptor-mediated gene transfer

C - Chemistry, Metallurgy

07

K

C07K 19/00 (2006.01) A61K 31/7088 (2006.01) A61K 47/48 (2006.01) A61K 48/00 (2006.01) C07H 21/00 (2006.01) C07K 14/81 (2006.01) C12N 15/87 (2006.01) C07K 14/705 (2006.01) C12N 9/64 (2006.01)

Patent

CA 2256558

Nucleic acids are compacted, substantially without aggregation, to facilitate their uptake by target cells of an organism to which the compacted material is administered. The nucleic acids may achieve a clinical effect as a result of gene expression, hybridization to endogenous nucleic acids whose expression is undesired, or site-specific integration so that a target gene is replaced, modified or deleted. The targeting may be enhanced by means of a target cell- binding moiety. The nucleic acid is preferably compacted to a condensed state.

L'invention concerne la compression d'acides nucléiques, sensiblement sans agrégation, pour faciliter le captage par des cellules cibles d'un organisme auquel on administre ce matériau comprimé. Les acides nucléiques peuvent produire un effet clinique suite à l'expression génique, à l'hybridation avec des acides nucléiques endogènes dont l'expression n'est pas souhaitée ou à un intégration dirigée sur site, de manière à remplacer, modifier ou supprimer un gène cible. On peut améliorer le ciblage au moyen d'un fraction susceptible d'être liée aux cellules cibles. De préférence, l'acide nucléique est comprimé jusqu'à un état condensé.

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