临床实践中的眼部基因治疗：病毒载体和非病毒替代物。
Ocular gene therapies in clinical practice: viral vectors and nonviral alternatives.
机构信息
Eyevensys S.A.S., Paris, France.
Inserm UMR_S 1138, Team 17, Centre de Recherche des Cordeliers, Paris, France; AP-HP Hôpitaux de Paris, Ophtalmopole Hôpital Cochin, Paris, France; Sorbonne University, University of Pierre et Marie Curie, UMR_S 1138, Centre de Recherche des Cordeliers, Paris, France; Paris Descartes University, Sorbonne Paris Cité, UMR_S 1138, Centre de Recherche des Cordeliers, Paris, France.
出版信息
Drug Discov Today. 2019 Aug;24(8):1685-1693. doi: 10.1016/j.drudis.2019.05.038. Epub 2019 Jun 5.
Ocular gene therapy has entered into clinical practice. Although viral vectors are currently the best option to replace and/or correct genes, the optimal method to deliver these treatments to the retinal pigment epithelial (RPE) cells and/or photoreceptor cells remains to be improved to increase transduction efficacy and reduce iatrogenic risks. Beyond viral-mediated gene replacement therapies, nonviral gene delivery approaches offer the promise of sustained fine-tuned expression of secreted therapeutic proteins that can be adapted to the evolving stage of the disease course and can address more common nongenetic retinal diseases, such as age-related macular degeneration (AMD). Here, we review current gene therapy strategies for ocular diseases, with a focus on clinical stage products.
眼基因治疗已进入临床实践。虽然病毒载体是目前替代和/或纠正基因的最佳选择，但将这些治疗方法递送至视网膜色素上皮（RPE）细胞和/或光感受器细胞的最佳方法仍有待改进，以提高转导效率并降低医源性风险。除了病毒介导的基因替代疗法外，非病毒基因传递方法有望持续微调分泌治疗蛋白的表达，这可以适应疾病进程的不断发展阶段，并可以解决更常见的非遗传性视网膜疾病，如年龄相关性黄斑变性（AMD）。在这里，我们综述了眼部疾病的当前基因治疗策略，重点是临床阶段产品。
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