Article
Preservation of photoreceptor morphology and function in P23H rats using an allele independent ribozyme.
Experimental eye research - 1 Jan 2007
Gorbatyuk M, Justilien V, Liu J, Hauswirth W W, Lewin A S
Abstract excerpt
To develop an allele independent ribozyme for the treatment of autosomal dominant retinitis pigmentosa (ADRP) associated with mutations in the rhodopsin (RHO) gene, a ribozyme targeting dog, mouse, human but not rat rhodopsin (RHO) mRNA was designed and tested in vitro. Activity of this ribozyme was tested in tissue culture by co-transfection of HEK 293 cells with plasmids expressing opsin mRNA and ribozyme,...
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