RS-1Gene Delivery to an AdultRs1hKnockout Mouse Model Restores ERG b-Wave with Reversal of the Electronegative Waveform of X-Linked Retinoschisis
Investigative Ophthalmology & Visual Science2004Vol. 45(9), pp. 3279–3279
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Yong Zeng, Yuichiro Takada, Sten Kjellström, Kelaginamane Hiriyanna, Atsuhiro Tanikawa, Eric F. Wawrousek, Nizar Smaoui, Rafael C. Caruso, Ronald A. Bush, Paul A. Sieving
Abstract
The RS-KO mouse mimics structural features of human X-linked juvenile retinoschisis with dissection through, and disorganization of, multiple retinal layers. The Rs1h-KO functional deficit results in an electronegative ERG waveform that is characteristic of human retinoschisis disease and that implicates a synaptic transmission deficit in the absence of retinoschisin protein. Replacement therapy by supplementing normal Rs1h protein in the adult Rs1h-KO mouse restored the normal ERG configuration. This indicates that gene therapy is a viable strategy of therapeutic intervention even in the postdevelopmental adult stage of XLRS disease.
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