in vivo effect of NRL Gene Silencing (KAP230672)
The final goal of this study is to develop a new concept treatment for age-related macular degeneration (AMD), which causes intractable blindness, through the development of in vivo retinal cell conversion technology. By knocking down NRL and ROR beta, which are key transcription factors for photoreceptor (PR) rod cell differentiation, which are concentrated in the central periphery of the macula, converting rods into cone-like cells (iCPR), improving central vision and preventing rod cell death. It is intended to establish a direct cross-differentiation technology in vivo that can mitigate.