Compacted nucleic acids and their delivery to cells
AssigneeCase Western Reserve University
InventorsRichard W. Hanson, Jose C. Perales, Thomas W. Ferkol, Jr.
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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.
Claims28
A method of preparing a composition comprising unaggregated nucleic acid complexes, each complex consisting essentially of a single nucleic acid molecule and one or more carrier molecules, said method comprising:
- mixing a nucleic acid molecule with a carrier molecule at a chaotropic salt concentration sufficient for compaction of the complex to a diameter which is less than 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, whereby unaggregated nucleic acid complexes are formed, wherein each complex consists essentially of a single nucleic acid molecule and one or more carrier molecules.
A method of preparing a composition comprising unaggregated nucleic acid complexes, each complex consisting essentially of a single nucleic acid molecule and one or more polycation molecules, said method comprising:
- mixing a nucleic acid molecule with a polycation in a solvent to form a complex, said mixing being performed in the absence of added chaotropic salt, whereby the nucleic acid forms soluble complexes with the polycation molecule without forming aggregates, wherein each complex consists essentially of a single nucleic acid molecule and one or more carrier molecules, wherein the complexes have a diameter which is less than 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.
Dependencies are inferred from the claim text.