Virus-mediated gene delivery is a very powerful tool for the treatment of inherited disorders (Bjorklund et al., 2000; Dejneka and Bennett, 2001; Manfredsson et al., 2006). Degenerations due to recessive mutations can be slowed or prevented by gene augmentation in which the wild-type form of the transgene of interest is packaged into a recombinant virus. Neurotrophic or neuroprotective factors can be delivered to tissues of interest by transduction with an appropriate virus. And, in the case of dominant inherited degenerations, a recombinant virus carrying siRNA specific to the mutant transgene could be introduced to the cells of interest to result in decreased production of the dominant negative form of the protein.
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Rex, T.S. (2008). Virus-mediated Gene Delivery to Neuronal Progenitors. 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_16
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