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重燃光明:治疗视网膜变性的创新策略

Rekindling Vision: Innovative Strategies for Treating Retinal Degeneration.

作者信息

Khan Irfan, Ramzan Faiza, Tayyab Haroon, Damji Karim F

机构信息

Department of Ophthalmology and Visual Sciences, The Aga Khan University, Stadium Road, P.O. Box 3500, Karachi 74800, Sindh, Pakistan.

Centre for Regenerative Medicine and Stem Cells Research, The Aga Khan University, Stadium Road, P.O. Box 3500, Karachi 74800, Sindh, Pakistan.

出版信息

Int J Mol Sci. 2025 Apr 25;26(9):4078. doi: 10.3390/ijms26094078.

Abstract

Retinal degeneration, characterized by the progressive loss of photoreceptors, retinal pigment epithelium cells, and/or ganglion cells, is a leading cause of vision impairment. These diseases are generally classified as inherited (e.g., retinitis pigmentosa, Stargardt disease) or acquired (e.g., age-related macular degeneration, diabetic retinopathy, glaucoma) ocular disorders that can lead to blindness. Available treatment options focus on managing symptoms or slowing disease progression and do not address the underlying causes of these diseases. However, recent advancements in regenerative medicine offer alternative solutions for repairing or protecting degenerated retinal tissue. Stem and progenitor cell therapies have shown great potential to differentiate into various retinal cell types and can be combined with gene editing, extracellular vesicles and exosomes, and bioactive molecules to modulate degenerative cellular pathways. Additionally, gene therapy and neuroprotective molecules play a crucial role in enhancing the efficacy of regenerative approaches. These innovative strategies hold the potential to halt the progression of retinal degenerative disorders, repair or replace damaged cells, and improve visual function, ultimately leading to a better quality of life for those affected.

摘要

视网膜变性以光感受器、视网膜色素上皮细胞和/或神经节细胞的进行性丧失为特征,是视力损害的主要原因。这些疾病通常被归类为遗传性(如色素性视网膜炎、斯特格氏病)或获得性(如年龄相关性黄斑变性、糖尿病性视网膜病变、青光眼)眼部疾病,可导致失明。现有的治疗方案侧重于控制症状或减缓疾病进展,并未解决这些疾病的根本原因。然而,再生医学的最新进展为修复或保护退化的视网膜组织提供了替代解决方案。干细胞和祖细胞疗法已显示出分化为各种视网膜细胞类型的巨大潜力,并且可以与基因编辑、细胞外囊泡和外泌体以及生物活性分子相结合,以调节退化的细胞途径。此外,基因疗法和神经保护分子在提高再生方法的疗效方面发挥着关键作用。这些创新策略有可能阻止视网膜退行性疾病的进展,修复或替换受损细胞,并改善视觉功能,最终为受影响的人带来更高的生活质量。

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