Serpin enzyme complex receptor--mediated gene transfer

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424 9321, 435 78, 435 914, 4353201, 435325, 435455, 53038822, 5308873, 530324, 536 231, A01N 4304

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059729015

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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