Gene therapy for inherited retinal degeneration

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Abstract
The eye has a number of advantages as a target organ for gene delivery. It is easily accessible and the tissues may be examined in vivo by ophthalmoscopy. In addition, there are blood-retinal and blood-aqueous barriers which may concentrate vectors in the target area and reduce their spread out of the eye. The eye may be used for testing gene delivery to a wide range of tissues since it contains endothelium (cornea), epithelium (cornea, ciliary body, iris), muscle (ciliary body), and neurons (retina). It may also serve as a valuable model system to test gene therapy strategies for the brain, whose neurons are more difficult to target than those in the neuroretina.