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Research Article

纳米增强光学基因传递到视网膜变性小鼠。

卷 19, 期 5, 2019

页: [318 - 329] 页: 12

弟呕挨: 10.2174/1566523219666191017114044

价格: $65

摘要

背景：将基因和其他不可渗透的治疗分子有效且有针对性地输送到视网膜细胞中对于治疗各种视觉障碍极为重要。用于基因递送的传统方法需要病毒转染，或具有一个或多个缺点（例如效率低，缺乏空间靶向递送）且通常具有有害作用（例如意外的炎症反应和免疫反应）的化学方法。 方法：我们旨在开发一种基于连续波近红外激光的纳米增强光学传递（NOD）方法，用于将环境光可激活的多特征视蛋白编码基因在空间内传递至视网膜内和离体。 。在这种方法中，利用金纳米棒增强的光场来瞬时透化细胞膜，从而能够将外源不可渗透分子递送到激光照射区域中的纳米棒结合细胞。 结果与讨论：通过病毒或其他非病毒（例如电穿孔，脂质转染）方法，基因无处不在，导致整个视网膜的表达不受控制。这将导致视网膜非变性区域的功能复杂化。在NOD方法中，纳米热点处的激光辐照附着纳米棒的细胞的温度升高对比度非常显着，足以实现大型基因的位点特异性传递。使用NOD进行的体内和体外结果清楚地证明了靶向基因的视网膜区域中的体内基因传递和功能性细胞表达，而不会损害眼睛的结构完整性或引起免疫反应。 结论：视网膜变性小鼠体内NOD后，在目标视网膜中MCO的成功递送和表达为视网膜萎缩症视网膜干性黄斑变性等视网膜萎缩再光敏化开辟了新的前景。

关键词: 眼基因治疗，光传递，光遗传学，干性AMD，黄斑变性，NOD方法。

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图形摘要

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