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AMD 与基于干细胞的疗法。

AMD and Stem Cell-Based Therapies.

出版信息

Int Ophthalmol Clin. 2024 Jan 1;64(1):21-33. doi: 10.1097/IIO.0000000000000510. Epub 2023 Dec 26.

Abstract

Age-related macular degeneration (AMD) is a prevalent and complex disease leading to severe vision loss. Stem cells offer promising prospects for AMD treatment as they can be differentiated into critical retinal cell types that could replace lost host retinal cells or provide trophic support to promote host retinal cell survival. However, challenges such as immune rejection, concerns regarding tumorigenicity, and genomic integrity must be addressed. Clinical trials with stem cell-derived retinal pigment epithelial cells have shown preliminary safety in treating dry AMD, but improvements in manufacturing and surgical techniques cell delivery are needed. Late-stage AMD poses additional hurdles, possibly requiring multi-layered grafts. Advancements in automation technologies and gene correction strategies show potential to enhance iPSC-based therapies. Stem cell-based treatments offer hope for AMD management, but further research and optimization are essential for successful clinical implementation.

摘要

年龄相关性黄斑变性(AMD)是一种常见且复杂的疾病,可导致严重的视力丧失。干细胞为 AMD 的治疗提供了有前景的前景,因为它们可以分化为关键的视网膜细胞类型,这些细胞类型可以替代丢失的宿主视网膜细胞,或提供营养支持以促进宿主视网膜细胞的存活。然而,必须解决免疫排斥、致瘤性和基因组完整性等方面的挑战。使用干细胞衍生的视网膜色素上皮细胞的临床试验在治疗干性 AMD 方面显示出初步的安全性,但需要改进制造和手术技术细胞输送。晚期 AMD 带来了额外的障碍,可能需要多层移植物。自动化技术和基因校正策略的进步显示出增强基于 iPSC 的治疗的潜力。基于干细胞的治疗为 AMD 的管理提供了希望,但为了成功的临床应用,还需要进一步的研究和优化。

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