RTP801 Gene Expression Is Differentially Upregulated in Retinopathy and Is Silenced by PF-04523655, a 19-Mer siRNA Directed Against RTP801

Abstract

In rodent retinopathy models, administration of the siRNA, PF-04523655, reduced RTP801 expression in the retina, consistent with the RNA-induced silencing complex (RISC) mechanism of action. The pharmacodynamic profile from the animal models could be useful to elucidate dose and exposure dependency of RTP801 expression inhibition by siRNA.