Nguyen Huy V, Li Yao, Tsang Stephen H
College of Physicians and Surgeons, Columbia University, 100 Haven Ave, Apt 14B, New York, NY 10032, USA.
Department of Ophthalmology, Columbia University, 635 W 165th St, New York, NY 10032, USA.
J Clin Med. 2015 Mar 31;4(4):567-78. doi: 10.3390/jcm4040567.
Inherited retinal diseases, such as age-related macular degeneration and retinitis pigmentosa, are the leading cause of blindness in the developed world. Currently, treatments for these conditions are limited. Recently, considerable attention has been given to the possibility of using patient-specific induced pluripotent stem cells (iPSCs) as a treatment for these conditions. iPSCs reprogrammed from adult somatic cells offer the possibility of generating patient-specific cell lines in vitro. In this review, we will discuss the current literature pertaining to iPSC modeling of retinal disease, gene therapy of iPSC-derived retinal pigmented epithelium (RPE) cells, and retinal transplantation. We will focus on the use of iPSCs created from patients with inherited eye diseases for testing the efficacy of gene or drug-based therapies, elucidating previously unknown mechanisms and pathways of disease, and as a source of autologous cells for cell replacement.
遗传性视网膜疾病，如年龄相关性黄斑变性和视网膜色素变性，是发达国家失明的主要原因。目前，针对这些病症的治疗方法有限。最近，使用患者特异性诱导多能干细胞（iPSC）作为这些病症的治疗方法受到了广泛关注。从成人体细胞重编程而来的iPSC提供了在体外生成患者特异性细胞系的可能性。在这篇综述中，我们将讨论与视网膜疾病的iPSC建模、iPSC衍生的视网膜色素上皮（RPE）细胞的基因治疗以及视网膜移植相关的当前文献。我们将重点关注利用患有遗传性眼病的患者所产生的iPSC来测试基于基因或药物的疗法的疗效、阐明先前未知的疾病机制和途径，以及作为细胞替代的自体细胞来源。
