COMPOSITIONS AND METHODS FOR EFFICACIOUS AND SAFE DELIVERY OF siRNA USING SPECIFIC CHITOSAN-BASED NANOCOMPLEXES
AssigneePolyvalor, Limited Partnership
InventorsAbderrazzak Merzouki, Michael D. Buschmann
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Abstract
There is disclosed a composition and a method for the efficient delivery of a therapeutic RNAi-inducing nucleic acid to cells both in vitro and in vivo through specific formulations of a non viral delivery system using chitosans. Particularly, the composition contains a nucleic acid and a specific chitosan that has the following physico-chemical properties: a number-average molecular weight between 5 kDa and 200 kDa, a degree of deacetylation between 80% and 95% and a chitosan amine to nucleic acid phosphate ratio below 20.
Claims31
A composition for inhibiting a gene expression in vivo comprising chitosan and an RNAi-inducing nucleic acid sequence against said gene wherein the chitosan has a molecular weight (Mn) of 5 kDa to 200 kDa, a degree of deacetylation (DDA) of 80% to 95%, and wherein the chitosan amine to nucleic acid phosphate ratio (N:P) is below 20.
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The method of claim 55, for treating diabetes mellitus related conditions wherein said diabetes mellitus related conditions are insulin-dependent diabetes mellitus (type I diabetes), noninsulin-dependent diabetes mellitus (type II diabetes), insulin resistance, hyperinsulinemia, diabetes-induced hypertension, obesity, damage to blood vessels, damage to eyes, damage to kidneys, damage to nerves, damage to autonomic nervous system, damage to skin, damage to connective tissue, and damage to immune system.
The method of claim 55, for treating atherosclerosis related conditions wherein said atherosclerosis related conditions are cardiovascular diseases.
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Dependencies are inferred from the claim text.