Nucleic acid delivery system
OwnerHolly G Blum, Per Arturssoninventors
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Abstract
The present invention is directed to a composition and pharmaceutical preparations for introducing nucleic acids including oligo- or poly-nucleotide into cells in a host tissue by a delivery system and a method of preparing such a composition. The composition for delivery of nucleic acids comprises polymeric carrier particles that are essentially free of groups having a positive electric charge and the nucleic acids are provided essentially on the surface of the particles. The carrier particle is insoluble in water but suitably it is able to absorb water quickly.
Claims47
A composition comprising polymeric carrier particles and nucleic acid, wherein the polymeric carrier particle is comprised of one or more polymers, is essentially insoluble in body fluids and aqueous solutions at a pH less than 8;
- and wherein further essentially each polymeric particle carries at least one nucleic acid essentially in a non-condensed form and associated therewith in such a way that at least part of said nucleic acid is positioned on the surface of the polymeric particle.
The composition of any preceding claim, wherein said coding sequence encodes a biologically active product.
The composition of any preceding claim, wherein the nucleic acid is associated with the polymeric particle in such a way that at least 50 percent by weight of the nucleic acid is positioned within 25 percent by volume of the peripheral parts of the polymeric particle.
The composition of any preceding claim, wherein the polymeric particles have a capacity to absorb an aqueous solution in an amount of at least fifty percent in relation to their own weight.
The composition of any preceding claim, wherein the polymeric particles comprise polymers selected from the group consisting of natural polymers, modified natural polymers, synthetic polymers, and mixtures thereof.
The composition of any preceding claim, wherein said polymer(s) are comprised of aggregates comprising at least 100 sub-units.
The composition of any preceding claim, wherein essentially 90% by weight of said polymeric particles have a size smaller than 200 μm, suitably smaller than 100 μm, and preferably smaller than 60 μm.
The composition of any preceding claim, wherein the ratio of positive electric charges and negative electric charges is such that the composition has an essentially net negative charge.
The composition of any preceding claim, wherein the polymeric carrier particle is essentially insoluble in mucosal fluid.
The composition of any preceding claim, wherein said composition further comprises at least one stabilizing agent such as a cryoprotectant.
A pharmaceutical preparation comprising the composition of any preceding claim in a physiologically administrable form.
A method of using polymeric carrier particles, which particles are comprised of one or more polymers, and are essentially insoluble in body fluids and aqueous solutions at a pH less than 8, to introduce nucleic acid or a polynucleotide into a cell, whereby said nucleic acid or polynucleotide is capable of expressing its function inside said cell.
Dependencies are inferred from the claim text.