Compacted nucleic acids and their delivery to cells

Details Compacted nucleic acids and their delivery to cells Compacted nucleic acids and their delivery to cells

C - Chemistry, Metallurgy

12

N

C12N 15/87 (2006.01) A61K 31/70 (2006.01) A61K 47/48 (2006.01) A61K 48/00 (2006.01) C07H 21/00 (2006.01) C07K 14/705 (2006.01) C07K 16/18 (2006.01) C07K 19/00 (2006.01) C12N 9/64 (2006.01) C12N 15/88 (2006.01)

Patent

CA 2186118

Nucleic acids are compared, 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 effet 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.

Des acides nucléiques sont compactés, pratiquement sans formation d'agrégats, pour faciliter leur capture par les cellules cibles d'un organisme auquel le matériel compacté a été administré. Ces acides nucléiques peuvent produire un effet clinique résultant de l'expression de gènes ou de leur hybridation en acides nucléiques endogènes dont l'expression est non désirée, ou s'intégrer spécifiquement à des sites de façon à remplacer, modifier ou détruire un gène cible. Le ciblage peut être renforcé à l'aide d'une fraction fixatrice de cellules d'une cible. L'acide nucléique est de préférence compacté jusqu'à l'état condensé.

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