Nuffield Laboratory of Ophthalmology, Department of Clinical Neurosciences, University of Oxford, Wellington Square, Oxford, OX1 2JD, UK.
Oxford University Hospital NIHR Biomedical Research Centre, Oxford, UK.
Mol Diagn Ther. 2024 Sep;28(5):575-591. doi: 10.1007/s40291-024-00722-0. Epub 2024 Jul 2.
Gene therapies have emerged as promising treatments in clinical development for various retinal disorders, offering hope to patients with inherited degenerative eye conditions. Several gene therapies have already shown remarkable success in clinical trials, with significant improvements observed in visual acuity and the preservation of retinal function. A multitude of gene therapies have now been delivered safely in human clinical trials for a wide range of inherited retinal disorders but there are some gaps in the reported trial data. Some of the most exciting treatment options are not under peer review and information is only available in press release form. Whilst many trials appear to have delivered good outcomes of safety, others have failed to meet primary endpoints and therefore not proceeded to phase III. Despite this, such trials have enabled researchers to learn how best to assess and monitor patient outcomes, which will guide future trials to greater success. In this review, we consider recent and ongoing clinical trials for a variety of potential retinal gene therapy treatments and discuss the positive and negative issues related to these trials. We discuss the treatment potential following clinical trials as well as the potential risks of some treatments under investigation. As these therapies continue to advance through rigorous testing and regulatory approval processes, they hold the potential to revolutionise the landscape of retinal disorder treatments, providing renewed vision and enhancing the quality of life for countless individuals worldwide.
基因疗法已成为各种视网膜疾病临床开发中很有前途的治疗方法，为遗传性退行性眼病患者带来了希望。几种基因疗法已经在临床试验中取得了显著的成功，观察到视力和视网膜功能的显著改善。目前已经有许多基因疗法在人体临床试验中安全地用于治疗广泛的遗传性视网膜疾病，但报告的试验数据仍存在一些空白。一些最令人兴奋的治疗选择未经过同行评审，信息仅以新闻稿的形式提供。虽然许多试验似乎都取得了良好的安全结果，但其他试验未能达到主要终点，因此未进入 III 期。尽管如此，这些试验使研究人员能够了解如何最好地评估和监测患者的结果，这将为未来的试验提供更大的成功指导。在这篇综述中，我们考虑了各种潜在的视网膜基因治疗方法的最新和正在进行的临床试验，并讨论了这些试验相关的积极和消极问题。我们讨论了临床试验后的治疗潜力以及正在研究的一些治疗方法的潜在风险。随着这些疗法在严格的测试和监管审批过程中不断推进，它们有可能彻底改变视网膜疾病治疗的格局，为全球无数人带来新的视力和提高生活质量。
