Autosomal Dominant Retinitis Pigmentosa (ADRP) is caused by mutations in the rhodopsin gene (RHO) in 30% of patients, and dominant negative mutations are often associated with toxicity of the mutated protein. Preventing or retarding ADRP progression may require the repair or silencing of the defective RHO gene. Two strategies for RHO mRNA silencing have recently been proposed (Millington-Ward et al., 1997; Lewin and Hauswirth, 2001). They are based on the application of therapeutic molecules such as ribozymes (Rz) and small interfering RNA (siRNA) that inhibit RHO expression.
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Gorbatyuk, M.S., Hauswirth, W.W., Lewin, A.S. (2008). Gene Therapy for Mouse Models of ADRP. 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_11
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