O'Brien Justine, Colucci Patrizia, Alvarez Yolanda, Kennedy Breandán N
UCD Conway Institute of Biomolecular and Biomedical Research, University College Dublin, Dublin, Ireland.
UCD School of Biomolecular and Biomedical Science, University College Dublin, Dublin, Ireland.
Adv Exp Med Biol. 2025;1468:491-495. doi: 10.1007/978-3-031-76550-6_80.
Photoreceptor and retinal pigment epithelium (RPE) dysfunction in inherited retinal degenerations (IRDs) and age-related macular degeneration (AMD) necessitate innovative therapies to preserve vision. Vision impairment incurs a substantial global economic burden, with the World Health Organization reporting an annual global productivity loss of approximately $411 billion. Current treatments are limited, underscoring the urgency for novel solutions. Leveraging new screening techniques, novel drugs restoring vision can be uncovered. Here, a workflow is described utilising orthogonal pooling to screen randomised library compounds for drug hits restoring vision and assessing the optokinetic response (OKR) in the atp6v0e1 zebrafish model of inherited blindness.
遗传性视网膜变性（IRD）和年龄相关性黄斑变性（AMD）中的光感受器及视网膜色素上皮（RPE）功能障碍，需要创新疗法来保护视力。视力损害给全球带来了巨大的经济负担，世界卫生组织报告称，全球每年的生产力损失约为4110亿美元。目前的治疗方法有限，凸显了寻找新解决方案的紧迫性。利用新的筛选技术，可以发现恢复视力的新型药物。在此，描述了一种工作流程，利用正交池化筛选随机文库化合物以寻找恢复视力的药物靶点，并在atp6v0e1遗传性失明斑马鱼模型中评估视动反应（OKR）。
