title: Polymeric vectors for ocular gene delivery.
abstract: Gene therapy holds promise for the treatment of many inherited and acquired diseases of the eye. Successful ocular gene therapy interventions depend on efficient gene transfer to targeted cells with minimal toxicity. A major challenge is to overcome both intracellular and extracellular barriers associated with ocular gene delivery. Numerous viral and nonviral vectors were explored to improve transfection efficiency. Among nonviral delivery systems, polymeric vectors have gained significant attention in recent years owing to their nontoxic and non-immunogenic nature. Polyplexes or nanoparticles can be prepared by interaction of cationic polymers with DNA, which facilitate cellular uptake, endolysosomal escape and nuclear entry through active mechanisms. Chemical modification of these polymers allows for the generation of flexible delivery vectors with desirable properties. In this article several synthetic and natural polymeric systems utilized for ocular gene delivery are discussed.
authors.affiliation: Division of Pharmaceutical Sciences, School of Pharmacy, University of Missouri-Kansas City, 2464 Charlotte Street, Kansas City, MO 64108-2718, USA.
authors.forename: Viral
authors.initials: V
authors.lastname: Tamboli
authors.forename: Gyan P
authors.initials: GP
authors.lastname: Mishra
authors.forename: Ashim K
authors.initials: AK
authors.lastname: Mitrat
mesh_terms: D060433:Administration, Ophthalmic; D000818:Animals; D001813:Blood-Retinal Barrier / Q000378:metabolism; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D053758:Nanoparticles / Q000008:administration & dosage / Q000737:chemistry / Q000627:therapeutic use; D011108:Polymers / Q000008:administration & dosage* / Q000737:chemistry / Q000378:metabolism / Q000627:therapeutic use; D014822:Vitreous Body / Q000378:metabolism
chemical_list: D011108:Polymers