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Retinal Pigment Epithelium Replacement Therapy for Age-Related Macular Degeneration: Are We There Yet?
Annual Review of Pharmacology and Toxicology ( IF 12.5 ) Pub Date : 2020-01-08 , DOI: 10.1146/annurev-pharmtox-010919-023245
Ruchi Sharma 1 , Devika Bose 1 , Arvydas Maminishkis 2 , Kapil Bharti 1
Affiliation  

Pluripotent stem cells (PSCs) are a potential replacement tissue source for degenerative diseases. Age-related macular degeneration (AMD) is a blinding disease triggered by degeneration of the retinal pigment epithelium (RPE), a monolayer tissue that functionally supports retinal photoreceptors. Recently published clinical and preclinical studies have tested PSC-derived RPE as a potential treatment for AMD. Multiple approaches have been used to manufacture RPE cells, to validate them functionally, to confirm their safety profile, and to deliver them to patients either as suspension or as a monolayer patch. Since most of these studies are at an early regulatory approval stage, the primary outcome has been to determine the safety of RPE transplants in patients. However, preliminary signs of efficacy were observed in a few patients. Here, we review the current progress in the PSC-derived RPE transplantation field and provide a comparative assessment of various approaches under development as potential therapeutics for AMD.

中文翻译:

年龄相关性黄斑变性的视网膜色素上皮替代疗法:我们还在那里吗?

多能干细胞 (PSC) 是退行性疾病的潜在替代组织来源。年龄相关性黄斑变性 (AMD) 是一种由视网膜色素上皮 (RPE) 变性引发的致盲疾病,视网膜色素上皮 (RPE) 是一种在功能上支持视网膜感光细胞的单层组织。最近发表的临床和临床前研究已经测试了 PSC 衍生的 RPE 作为 AMD 的潜在治疗方法。已使用多种方法来制造 RPE 细胞,对其功能进行验证,确认其安全性,并将它们作为悬浮液或单层贴片交付给患者。由于大多数这些研究处于早期监管批准阶段,主要结果是确定患者 RPE 移植的安全性。然而,在少数患者中观察到初步的疗效迹象。这里,
更新日期:2020-04-21
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