Article
Development of lead hammerhead ribozyme candidates against human rod opsin mRNA for retinal degeneration therapy.
Experimental eye research - 1 May 2009
Abdelmaksoud Heba E, Yau Edwin H, Zuker Michael, Sullivan Jack M
Abstract excerpt
To identify lead candidate allele-independent hammerhead ribozymes (hhRz) for the treatment of autosomal dominant mutations in the human rod opsin (RHO) gene, we tested a series of hhRzs for potential to significantly knockdown human RHO gene expression in a human cell expression system. Multiple computational criteria were used to select target mRNA regions likely to be single stranded and accessible to hhRz...
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