Mutation–Independent suppression of rhodopsin in Autosomal dominant Retinitis Pigmentosa by siRNA.

Abstract

Abstract: : Purpose: To monitor suppression levels of human rhodopsin transcript treated with a range of siRNAs and compare it to expression levels of a replacement rhodopsin gene with altered target sites. Methods: Cos7 cells expressing human rhodopsin were treated with siRNA. Transcript RNA and protein levels were assessed by quantitative rt–PCR and western blotting respectively. Results: Wild–type rhodopsin transcript can be suppressed without affecting expression of an altered rhodopsin message that codes for the same amino acids but has some alternative degenerate base–pair target sites. Conclusions: We have identified agents with potential as suppression components in a suppression and replacement therapeutic strategy.

Keywords: retinal degenerations: hereditary • gene/expression • gene transfer/gene therapy