Kevin Y Wu, Jaskarn K Dhaliwal, Akash Sasitharan, Ananda Kalevar
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引用次数: 0
摘要
背景/目标:老年性黄斑变性(AMD)和视网膜色素变性(RP)是导致视力丧失的主要原因,其中老年性黄斑变性主要影响老年人群,而视网膜色素变性则是一种较罕见的遗传性疾病。这两种疾病都会导致渐进性视网膜变性,而目前的治疗方法在晚期仍无法解决这一问题。本综述旨在概述视网膜的解剖学和生理学,阐明 AMD 和 RP 的病理生理学,并评估针对这些疾病的新兴细胞疗法。方法:对文献进行了全面回顾,重点关注细胞疗法,包括胚胎干细胞(ESC)、诱导多能干细胞(iPSC)、间充质干细胞(MSC)和视网膜祖细胞。对临床前和临床研究进行了分析,以评估治疗潜力,并关注细胞替代、神经保护和旁分泌效应等机制。此外,还探讨了相关挑战,包括伦理问题和临床转化。结果:细胞疗法通过神经保护和细胞替代等机制,显示出恢复视网膜功能和延缓疾病进展的潜力。临床前试验显示了良好的结果,但临床研究面临着巨大的障碍,包括细胞输送和长期疗效方面的挑战。整合基因编辑和生物材料的组合疗法有望在未来取得进展。结论:基于细胞的治疗 AMD 和 RP 取得了重大进展,但临床应用仍面临巨大障碍。要克服这些障碍、改进给药方法并确保这些疗法安全有效地应用于临床实践,就必须开展进一步的研究。
Cell Therapy for Retinal Degenerative Diseases: Progress and Prospects.
Background/Objectives: Age-related macular degeneration (AMD) and retinitis pigmentosa (RP) are leading causes of vision loss, with AMD affecting older populations and RP being a rarer, genetically inherited condition. Both diseases result in progressive retinal degeneration, for which current treatments remain inadequate in advanced stages. This review aims to provide an overview of the retina's anatomy and physiology, elucidate the pathophysiology of AMD and RP, and evaluate emerging cell-based therapies for these conditions. Methods: A comprehensive review of the literature was conducted, focusing on cell therapy approaches, including embryonic stem cells (ESCs), induced pluripotent stem cells (iPSCs), mesenchymal stem cells (MSCs), and retinal progenitor cells. Preclinical and clinical studies were analyzed to assess therapeutic potential, with attention to mechanisms such as cell replacement, neuroprotection, and paracrine effects. Relevant challenges, including ethical concerns and clinical translation, were also explored. Results: Cell-based therapies demonstrate potential for restoring retinal function and slowing disease progression through mechanisms like neuroprotection and cell replacement. Preclinical trials show promising outcomes, but clinical studies face significant hurdles, including challenges in cell delivery and long-term efficacy. Combination therapies integrating gene editing and biomaterials offer potential future advancements. Conclusions: While cell-based therapies for AMD and RP have made significant progress, substantial barriers to clinical application remain. Further research is essential to overcome these obstacles, improve delivery methods, and ensure the safe and effective translation of these therapies into clinical practice.
PharmaceuticsPharmacology, Toxicology and Pharmaceutics-Pharmaceutical Science
CiteScore
7.90
自引率
11.10%
发文量
2379
审稿时长
16.41 days
期刊介绍:
Pharmaceutics (ISSN 1999-4923) is an open access journal which provides an advanced forum for the science and technology of pharmaceutics and biopharmaceutics. It publishes reviews, regular research papers, communications, and short notes. Covered topics include pharmacokinetics, toxicokinetics, pharmacodynamics, pharmacogenetics and pharmacogenomics, and pharmaceutical formulation. Our aim is to encourage scientists to publish their experimental and theoretical details in as much detail as possible. There is no restriction on the length of the papers. The full experimental details must be provided so that the results can be reproduced.