초록 ▼

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 e

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.

대표청구항 ▼

[ We claim:] [1.] A non-naturally occurring composition comprising unaggregated nucleic acid complexes, each complex consisting essentially of a single nucleic acid molecule and one or more carrier molecules, said carrier molecule having a nucleic acid binding moiety through which it is complexed to

[ We claim:] [1.] A non-naturally occurring composition comprising unaggregated nucleic acid complexes, each complex consisting essentially of a single nucleic acid molecule and one or more carrier molecules, said carrier molecule having a nucleic acid binding moiety through which it is complexed to the nucleic acid, and a target cell-binding moiety through which it may bind to a target cell and whereby the complex may more readily enter the target cell, wherein said complex is compacted to a diameter which is less than (a) double the theoretical minimum diameter of a complex of said single nucleic acid molecule and a sufficient number of carrier molecules to provide a charge ratio of 1:1, in the form of a condensed sphere, or (b) 30 nm, whichever is larger.