Compacted nucleic acids and their delivery to cells

Organic compounds -- part of the class 532-570 series – Organic compounds – Carbohydrates or derivatives

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424493, 424 9321, 514 44, C12N 1511

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active

060083360

ABSTRACT:
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.

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