Stephen A. Wynn Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, USA.
Stephen A. Wynn Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, USA; Howard Hughes Medical Institute, University of Iowa, Iowa City, IA, USA.
Prog Retin Eye Res. 2015 Jan;44:15-35. doi: 10.1016/j.preteyeres.2014.10.002. Epub 2014 Nov 4.
Vision is the sense that we use to navigate the world around us. Thus it is not surprising that blindness is one of people's most feared maladies. Heritable diseases of the retina, such as age-related macular degeneration and retinitis pigmentosa, are the leading cause of blindness in the developed world, collectively affecting as many as one-third of all people over the age of 75, to some degree. For decades, scientists have dreamed of preventing vision loss or of restoring the vision of patients affected with retinal degeneration through drug therapy, gene augmentation or a cell-based transplantation approach. In this review we will discuss the use of the induced pluripotent stem cell technology to model and develop various treatment modalities for the treatment of inherited retinal degenerative disease. We will focus on the use of iPSCs for interrogation of disease pathophysiology, analysis of drug and gene therapeutics and as a source of autologous cells for cell transplantation and replacement.
视觉是我们用于探索周围世界的感觉。因此，失明是人们最害怕的疾病之一也就不足为奇了。遗传性视网膜疾病，如年龄相关性黄斑变性和色素性视网膜炎，是发达国家失明的主要原因，在某种程度上，每三分之一的 75 岁以上人群都会受到影响。几十年来，科学家们一直梦想着通过药物治疗、基因增强或基于细胞的移植方法来预防视力丧失或恢复受视网膜变性影响的患者的视力。在这篇综述中，我们将讨论诱导多能干细胞技术在遗传性视网膜退行性疾病的各种治疗模式的建模和开发中的应用。我们将重点介绍 iPSCs 在疾病病理生理学研究、药物和基因治疗分析以及作为细胞移植和替代的自体细胞来源方面的应用。
