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.
Publication types
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Research Support, Non-U.S. Gov't
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Review
MeSH terms
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Animals
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Cattle
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Drug Carriers* / administration & dosage
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Drug Carriers* / adverse effects
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Drug Carriers* / chemistry
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Drug Stability
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Epithelial Cells / drug effects
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Epithelial Cells / metabolism
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Eye Diseases / therapy
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Gene Transfer Techniques*
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Genetic Therapy / methods
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Humans
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Liposomes
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Luciferases, Firefly / genetics
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Microbubbles
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Nanoparticles* / administration & dosage
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Nanoparticles* / adverse effects
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Nanoparticles* / chemistry
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Nucleic Acids / administration & dosage*
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Nucleic Acids / genetics
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Pigment Epithelium of Eye / cytology
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Polyethylene Glycols* / administration & dosage
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Polyethylene Glycols* / adverse effects
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Polyethylene Glycols* / chemistry
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Transfection
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Ultrasonics
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Vitreous Body / drug effects*
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Vitreous Body / metabolism
Substances
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Drug Carriers
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Liposomes
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Nucleic Acids
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Polyethylene Glycols
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Luciferases, Firefly