视网膜色素变性(RP)是一种进行性、可致盲的遗传性眼底病。抑制穆勒细胞(MC)胶质化，促进其重编程转分化为视网膜神经细胞是RP防治的重要途径。miR-let-7介导的Shh信号通路广泛参与调控RP进程中MC重编程及转分化环节。滋阴活血明目方剂丹杞明目汤(DQMMT)具有良好的改善RP患者视力与视野的临床疗效。本项目据此提出假说：DQMMT通过抑制MC胶质化并诱导其重编程转分化为视网膜神经细胞，从而改善视功能，治疗RP，其分子机制与调控miR-let-7介导的Shh信号通路有关。为证实该假说，本项目拟开展体内外实验和临床研究，采用ERG、OCT、免疫荧光、细胞共培养等方法，以视网膜形态、功能及神经细胞分化为评价指标，从基础与临床层面系统考察DQMMT诱导MC重编程治疗RP的效应及其通过调控miR-let-7介导的Shh信号通路的可能机制，为眼科临床运用DQMMT治疗RP的推广提供科学依据。

Retinitis pigmentosa (RP) is a progressive and hereditary retinal disease that can cause blindness. Recent evidence suggests that inhibition of Müller cell (MC) glialization and promotion of reprogramming of MC into retinal neurons are key aspects of prevention and treatment of RP. The miR-let-7-mediated Shh signaling pathway is widely involved in regulating MC reprogramming and transdifferentiation during RP. Dan-Qi-Ming-Mu-Tang (DQMMT), a TCM prescription for nourishing yin, promoting blood circulation, and improving eyesight, has a good clinical effect on improving the visual function of RP patients. We therefore propose the hypothesis that DQMMT improves the visual function and treats RP by inhibiting MC glialization and inducing it reprogramming into retinal nerve cells, and its molecular mechanism is associated with the regulation of miR-let-7-mediated Shh signaling cascade. In order to confirm this hypothesis, we intend to carry out in vivo and in vitro experiments and clinical research, using methods of ERG, OCT, immunofluorescence, cell co-culture, etc., taking retinal morphology and function and neuron differentiation as evaluation indexes. The effect of DQMMT on inducing MC reprogramming to treat RP and its possible mechanism of regulating the miR-let-7 mediated Shh signaling pathway are systematically investigated from the basic and clinical levels. The purpose of this project is to provide scientific basis for the promotion of clinical application of DQMMT for RP treatment in ophthalmology.