Article
A mutation-independent therapeutic strategem for osteogenesis imperfecta.
Antisense & nucleic acid drug development - 1 Dec 1999
Millington-Ward S, O'Neill B, Kiang A S, Humphries P, Kenna P F, Farrar G J
Abstract excerpt
Given the genetically heterogeneous nature of many dominantly inherited disorders, it will be imperative to design mutation-independent therapeutic strategies to circumvent such heterogeneity. Intragenic polymorphism represents a genomic resource that may be harnessed in the development of allele-specific mutation-independent therapeutics. A hammerhead ribozyme, Rzpol1a1, selectively cleaves a common...
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