Abstract
The eye is at the forefront of developing therapies for genetic diseases. With the FDA approval of the first gene-therapy drug for a form of congenital blindness, numerous studies have been initiated to develop gene therapies for other forms of eye diseases. These examinations have revealed new information about the benefits as well as restrictions to using drug-delivery routes to the different parts of the eye. In this article, we will discuss a brief history of gene therapy and its importance to the eye and ocular delivery landscape that is currently being investigated, and provide insights into their advantages and disadvantages. Efficient delivery routes and vehicle are crucial for an effective, safe, and longer-lasting therapy.
Keywords:
eye; gene therapy; retina.
Publication types
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Research Support, N.I.H., Extramural
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Review
MeSH terms
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Animals
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Choroidal Effusions
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DNA / genetics
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DNA / metabolism
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DNA / therapeutic use
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Eye / metabolism
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Eye / pathology
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Eye Diseases / genetics
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Eye Diseases / metabolism
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Eye Diseases / pathology
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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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Genetic Vectors / administration & dosage
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Genetic Vectors / chemistry
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Genetic Vectors / metabolism*
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Humans
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Intravitreal Injections
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Liposomes / chemistry
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Liposomes / metabolism
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Liposomes / therapeutic use
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Nanoparticles / administration & dosage
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Nanoparticles / chemistry
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Nanoparticles / metabolism
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Peptides / chemistry
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Peptides / metabolism
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Peptides / therapeutic use
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Subretinal Fluid
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Viruses / genetics
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Viruses / metabolism
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Vitreous Body