Dually derivatized chitosan nanoparticles and methods of making and using the same for gene transfer in vivo
First Claim
1. A method of treating diabetes in a patient in need thereof, the method comprising administering a therapeutically effective amount of a therapeutic nucleic acid encoding insulin, a glucagon antagonist, GLP-1 or leptin 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 said chitosan-derivative nanoparticle comprises chitosan functionalized with arginine (Arg) and a hydrophilic polyol (HP) of Formula VII:
1 Assignment
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Accused Products
Abstract
Provided herein is chitosan-derivative nanoparticle comprising chitosan functionalized with a cationic amino acid and a hydrophilic polyol; and methods of making and using same, e.g., for gene delivery in vivo.
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Citations
10 Claims
- 1. A method of treating diabetes in a patient in need thereof, the method comprising administering a therapeutically effective amount of a therapeutic nucleic acid encoding insulin, a glucagon antagonist, GLP-1 or leptin 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 said chitosan-derivative nanoparticle comprises chitosan functionalized with arginine (Arg) and a hydrophilic polyol (HP) of Formula VII:
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2. A method of treating inflammatory bowel disease in a patient in need thereof, the method comprising administering a therapeutically effective amount of a therapeutic nucleic acid encoding IL-10, a TNFα
- antagonist, or an IL-17 antagonist 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 said chitosan-derivative nanoparticle comprises chitosan functionalized with arginine (Arg) and a hydrophilic polyol (HP) of Formula VII;
- antagonist, or an IL-17 antagonist 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 said chitosan-derivative nanoparticle comprises chitosan functionalized with arginine (Arg) and a hydrophilic polyol (HP) of Formula VII;
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3. A method of treating of obesity in a patient in need thereof, the method comprising administering a therapeutically effective amount of a therapeutic nucleic acid encoding leptin, cholecystokinin, PYY or GLP-1 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 said chitosan-derivative nanoparticle comprises chitosan functionalized with arginine (Arg) and a hydrophilic polyol (HP) of Formula VII:
Specification