Article
Ribozyme-based therapeutic approaches for autosomal dominant retinitis pigmentosa.
Investigative ophthalmology & visual science - 1 Sept 2000
O'Neill B, Millington-Ward S, O'Reilly M, Tuohy G, Kiang A S, Kenna P F, Humphries P, Farrar G J
Abstract excerpt
PURPOSE: To design, generate, and compare in vitro a range of hammerhead ribozymes targeting retinal transcripts implicated in autosomal dominant retinitis pigmentosa (adRP) and thereby identify ribozymes that may be valuable as therapeutic agents for adRP. To address mutational heterogeneity in rhodopsin and peripherin-linked adRP using mutation-independent ribozyme-based therapeutic approaches. METHODS:...
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