PDF(Pubmed)
Abstract：
Retinitis pigmentosa and age-related macular degeneration are the most frequent causes of irreversible visual impairment in the world. Existing therapeutic methods could be more effective, underscoring the necessity of new treatments. Reconstructing the retinal photoreceptors through the transplantation of human pluripotent stem cells, representing an attractive approach for restoring vision, has gained momentum. This paper gives an exhaustive account of what has been known in this field, the discoveries made, and the recent progress. This review paper outlines the retina\'s organisation, cell types, the pathophysiology of retinal injury/degeneration, and the reasoning behind using pluripotent stem cells in retinal regeneration. This article investigates differentiation strategies, molecular components that dictate cell type specification, and the recreation of retinal development in vitro, genetically engineering and manipulating epigenetic marks using various techniques for driving specific cell fates and improving therapy efficacy. Subretinal injection methods, cell encapsulation techniques, scaffold-based approaches, cell sheet transplantation, and their impact on integrating implanted cells into a functional retina are thoroughly reviewed. Using bioengineering approaches, biomaterials and growth factors form a favourable micro-ambience for grafted cells. Issues around safety and efficacy (tumorigenicity, immunological rejection, and long-term integration/functionality) are explored. Moreover, the paper emphasises the significance of rigorous characterisation, immunomodulatory strategies, and clinical and pre-clinical studies to ensure the safety and effectiveness of retinal regeneration therapy. Future perspectives and challenges are presented, looking at fine-tuning differentiation strategies, improving functional integration and regulatory aspects, and using co-therapy and supportive treatments.
摘要：
视网膜色素变性和年龄相关性黄斑变性是世界上最常见的不可逆视力损害的原因。现有的治疗方法可能更有效，强调新疗法的必要性。通过移植人多能干细胞重建视网膜光感受器，代表了一种有吸引力的恢复视力的方法，获得了势头。本文详尽地介绍了这一领域的已知情况，这些发现,以及最近的进展。这篇综述论文概述了视网膜的组织，细胞类型，视网膜损伤/变性的病理生理学，以及在视网膜再生中使用多能干细胞的原因。本文研究了差异化策略，决定细胞类型规格的分子成分，和体外视网膜发育的娱乐，使用各种技术进行基因工程和操纵表观遗传标记，以驱动特定的细胞命运并提高治疗效果。视网膜下注射方法,细胞封装技术，基于支架的方法，细胞片移植,以及它们对将植入细胞整合到功能性视网膜中的影响进行了全面回顾。使用生物工程方法，生物材料和生长因子对移植细胞形成有利的微环境。围绕安全性和有效性的问题(致瘤性，免疫排斥,和长期集成/功能)进行了探索。此外，论文强调了严格表征的重要性，免疫调节策略，以及临床和临床前研究，以确保视网膜再生治疗的安全性和有效性。提出了未来的观点和挑战，着眼于微调差异化策略，改善功能整合和监管方面，并使用共同治疗和支持治疗。
