Du Hongjun, Lim Siok Lam, Grob Seanna, Zhang Kang
Institute for Genomic Medicine and Shiley Eye Center, University of California at San Diego, La Jolla, CA 92093, USA.
Semin Ophthalmol. 2011 May;26(3):216-24. doi: 10.3109/08820538.2011.577498.
There is currently no FDA-approved therapy for treating patients with geographic atrophy (GA), a late stage of age-related macular degeneration (AMD). Cell transplantation has the potential to restore vision in these patients. This review discusses how recent advancement in induced pluripotent stem (iPS) cells provides a promising therapy for GA treatment. Recent advances in stem cell biology have demonstrated that it is possible to derive iPS cells from human somatic cells by introducing reprogramming factors. Human retinal pigment epithelium (RPE) cells and photoreceptors can be derived from iPS cells by defined factors. Studies show that transplanting these cells can stabilize or recover vision in animal models. However, cell derivation protocols and transplantation procedures still need to be optimized. Much validation has to be done before clinical-grade, patient-derived iPS can be applied for human therapy. For now, RPE cells and photoreceptors derived from patient-specific iPS cells can serve as a valuable tool in elucidating the mechanism of pathogenesis and drug discovery for GA.
目前尚无美国食品药品监督管理局（FDA）批准的用于治疗地图样萎缩（GA）患者的疗法，GA是年龄相关性黄斑变性（AMD）的晚期阶段。细胞移植有可能恢复这些患者的视力。本综述讨论了诱导多能干细胞（iPS）的最新进展如何为GA治疗提供了一种有前景的疗法。干细胞生物学的最新进展表明，通过引入重编程因子，有可能从人类体细胞中获得iPS细胞。通过特定因子，人类视网膜色素上皮（RPE）细胞和光感受器可从iPS细胞中分化而来。研究表明，在动物模型中移植这些细胞可稳定或恢复视力。然而，细胞分化方案和移植程序仍需优化。在临床级、患者来源的iPS可应用于人类治疗之前，还必须进行大量验证。目前，源自患者特异性iPS细胞的RPE细胞和光感受器可作为阐明GA发病机制和药物发现的宝贵工具。
