Review: Retinal degeneration: Focus on the unfolded protein response
Review: Retinal degeneration: Focus on the unfolded protein response
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DOI：
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发表时间：
2013-09
期刊：
影响因子：
2.2
通讯作者：
M. Gorbatyuk;O. Gorbatyuk
中科院分区：
文献类型：
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作者：
M. Gorbatyuk;O. Gorbatyuk
最近发表的文献提供了证据表明，未折叠蛋白反应（UPR）参与视网膜变性的发展。这些研究的范围包括糖尿病视网膜病变、早产儿视网膜病变、青光眼、视网膜脱离、光诱导的视网膜变性、年龄相关性黄斑变性和遗传性视网膜变性。随后的研究调查的作用，个别的UPR标志物在视网膜发病机制和检查的治疗潜力的重新编程的UPR作为一种方法，用于调节视网膜变性的速度已经启动。操纵的UPR标记已成为可能，通过使用基因敲除小鼠，药理学试剂，和病毒载体介导的基因表达增强。未来的研究将致力于鉴定UPR调节标记物的特异性抑制剂和/或诱导剂，以及扩大UPR相关动物模型的列表。此外，腺相关病毒介导的基因递送是一种安全有效的调节基因表达的方法，因此是一种有用的研究工具，用于操纵受影响的视网膜中的单个UPR标记物，并且是一种有前途的用于视网膜变性疾病的基因治疗的递送载体。
Recently published literature has provided evidence that the unfolded protein response (UPR) is involved in the development of retinal degeneration. The scope of these studies encompassed diabetic retinopathy, retinopathy of prematurity, glaucoma, retinal detachment, light-induced retinal degeneration, age-related macular degeneration, and inherited retinal degeneration. Subsequent studies investigating the role of individual UPR markers in retinal pathogenesis and examining the therapeutic potential of reprogramming the UPR as a method for modulating the rate of retinal degeneration have been initiated. Manipulation of UPR markers has been made possible by the use of knockout mice, pharmacological agents, and viral vector-mediated augmentation of gene expression. Future research will aim at identifying specific inhibitors and/or inducers of UPR regulatory markers as well as expand the list of UPR-related animal models. Additionally, adeno-associated virus-mediated gene delivery is a safe and effective method for modulating gene expression, and thus is a useful research tool for manipulating individual UPR markers in affected retinas and a promising delivery vector for gene therapy in retinal degenerative disorders.