title: Wanted and unwanted properties of surface PEGylated nucleic acid nanoparticles in ocular gene transfer.
abstract: Ocular gene therapy may offer new hope for severe eye diseases. Many of these ocular diseases are due to a gene defect in the retina, a multi-layered sensory tissue that lines the back of the eye. However, it is well known that the blood-retina barrier and sclera prevent hydrophilic and high molecular weight drugs to reach the retina after systemic or topical application. Therefore, intravitreal injection of non-viral nucleic acid nanoparticles has been considered as a safe and promising approach in ocular gene transfer. However, after intravitreal injection the non-viral nucleic acid nanoparticles should be stable and mobile in the vitreous. In this overview we focus on the behavior of non-viral nucleic acid nanoparticles (lipoplexes) in vitreous and on PEGylation strategies that improve their behavior in vitreous, but that do not affect their transfection capacity.
authors.affiliation: Laboratory of General Biochemistry and Physical Pharmacy, Faculty of Pharmacy, Ghent University, Harelbekestraat 72, 9000 Ghent, Belgium. Niek.Sanders@UGent.be
authors.forename: Niek N
authors.initials: NN
authors.lastname: Sanders
authors.forename: Liesbeth
authors.initials: L
authors.lastname: Peeters
authors.forename: Ine
authors.initials: I
authors.lastname: Lentacker
authors.forename: Joseph
authors.initials: J
authors.lastname: Demeester
authors.forename: Stefaan C
authors.initials: SC
authors.lastname: De Smedt
mesh_terms: D000818:Animals; D002417:Cattle; D004337:Drug Carriers / Q000008:administration & dosage / Q000009:adverse effects / Q000737:chemistry; D004355:Drug Stability; D004847:Epithelial Cells / Q000187:drug effects / Q000378:metabolism; D005128:Eye Diseases / Q000628:therapy; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods; D006801:Humans; D008081:Liposomes; D049409:Luciferases, Firefly / Q000235:genetics; D045423:Microbubbles; D053758:Nanoparticles / Q000008:administration & dosage / Q000009:adverse effects / Q000737:chemistry; D009696:Nucleic Acids / Q000008:administration & dosage* / Q000235:genetics; D010857:Pigment Epithelium of Eye / Q000166:cytology; D011092:Polyethylene Glycols / Q000008:administration & dosage / Q000009:adverse effects / Q000737:chemistry; D014162:Transfection; D014465:Ultrasonics; D014822:Vitreous Body / Q000187:drug effects* / Q000378:metabolism
chemical_list: D004337:Drug Carriers; D008081:Liposomes; D009696:Nucleic Acids; D011092:Polyethylene Glycols; D049409:Luciferases, Firefly