Dually derivatized chitosan nanoparticles and methods of making and using the same for gene transfer in vivo
AssigneeenGene, Inc.
InventorsJun GAO, Eric HSU, Anthony CHEUNG
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Abstract
Provided herein is chitosan dually derivatized with arginine and gluconic acid; and methods of making and using the same, e.g., for gene delivery in vivo.
Claims22
1-14. (canceled)
A method for modulating an immune response in a patient in need thereof, the method comprising administering a therapeutically effective amount of a therapeutic nucleic acid to a target tissue in the patient, wherein said administering comprises contacting said target tissue with a dually derivatized (DD) chitosan nucleic acid polyplex, said DD chitosan nucleic acid polyplex comprising a chitosan-derivative nanoparticle and said therapeutic nucleic acid, wherein the chitosan-derivative nanoparticle comprises chitosan functionalized with arginine (Arg) and gluconic acid.
A dually derivatized-chitosan compound of Formula I wherein chitosan is coupled to arginine and gluconic acid:
- wherein n is an integer of 1 to 2000,
- α is the functionalization degree of arginine,
- β is the functionalization degree of gluconic acid;
- and
- each R1 is independently selected from hydrogen, acetyl, Formula (II), and Formula (III).
Dependencies are inferred from the claim text.