There are a variety of diseases of the retina arising from genetic and non-genetic causes, or a combination of both, that lead to the loss of vision. The retina is a prime location for gene therapy because of its accessibility, immune privileged status (Caspi, 2006), and susceptible cell types. Several strategies have been attempted to rescue retinal disease, including gene replacement (Acland et al., 2001), gene knockdown with both ribozymes (Gorbatyuk et al., 2007) and siRNA (Kiang et al., 2005), and therapeutic gene supplementation (Deng et al., 2005).
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Alexander, J.J., Hauswirth, W.W. (2008). Adeno-Associated Viral Vectors and the Retina. In: Anderson, R.E., LaVail, M.M., Hollyfield, J.G. (eds) Recent Advances in Retinal Degeneration. Advances in Experimental Medicine and Biology, vol 613. Springer, New York, NY. https://doi.org/10.1007/978-0-387-74904-4_13
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