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Near-Infrared Laser-Based Spatially Targeted Nano-enhanced Optical Delivery of Therapeutic Genes to Degenerated Retina.
Molecular Therapy - Methods & Clinical Development ( IF 4.6 ) Pub Date : 2020-04-07 , DOI: 10.1016/j.omtm.2020.03.030 Subrata Batabyal 1 , Sivakumar Gajjeraman 1 , Kissaou Tchedre 1 , Adnan Dibas 1 , Weldon Wright 1 , Samarendra Mohanty 1
Molecular Therapy - Methods & Clinical Development ( IF 4.6 ) Pub Date : 2020-04-07 , DOI: 10.1016/j.omtm.2020.03.030 Subrata Batabyal 1 , Sivakumar Gajjeraman 1 , Kissaou Tchedre 1 , Adnan Dibas 1 , Weldon Wright 1 , Samarendra Mohanty 1
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Non-viral delivery of therapeutic genes into targeted areas of retina is essential for re-functionalizing the retinal circuitry. While a focused ultrafast laser beam has been recently used for intra-ocular delivery of molecules, it poses the significant technical challenge of overcoming aberrations of the eye and maintaining a tightly focused spot on the retinal cell membrane. Furthermore, to minimize collateral damage and increase the throughput of gene delivery, we introduced a weakly focused near-infrared (NIR) continuous wave (CW) or pulsed laser beam on to the cells wherein the intensity is locally enhanced by gold nanorods bound to the cell membranes to permit gene insertion. Parametric optimization of nano-enhanced optical delivery (NOD) was carried out by varying the exposure time, as well as the power of the CW NIR beam or the energy of the pulsed NIR beam. Using this NOD method, therapeutic genes encoding for multi-characteristic opsins (MCOs) were delivered to spatially targeted regions of degenerated retina ex vivo as well as in vivo. NOD-mediated cell membrane-specific expression of MCOs in targeted retinal regions with photoreceptor degeneration will allow functional recovery in an ambient light environment.
中文翻译：
基于近红外激光的空间靶向纳米增强光学传输治疗基因至退化视网膜。
将治疗基因非病毒传递到视网膜的目标区域对于视网膜回路的重新功能化至关重要。虽然聚焦超快激光束最近已用于眼内分子输送，但它提出了克服眼睛像差并在视网膜细胞膜上保持紧密聚焦点的重大技术挑战。此外，为了最大限度地减少附带损害并提高基因传递的吞吐量，我们向细胞引入了弱聚焦近红外（NIR）连续波（CW）或脉冲激光束，其中与细胞结合的金纳米棒局部增强了强度。细胞膜允许基因插入。通过改变曝光时间以及 CW NIR 光束的功率或脉冲 NIR 光束的能量，对纳米增强光学传输 (NOD) 进行参数优化。使用这种 NOD 方法，编码多特征视蛋白 (MCO) 的治疗基因被递送到离体和体内退化视网膜的空间目标区域。 NOD 介导的 MCO 在光感受器变性的目标视网膜区域中的细胞膜特异性表达将允许在环境光环境中功能恢复。
更新日期：2020-04-07
中文翻译：
基于近红外激光的空间靶向纳米增强光学传输治疗基因至退化视网膜。
将治疗基因非病毒传递到视网膜的目标区域对于视网膜回路的重新功能化至关重要。虽然聚焦超快激光束最近已用于眼内分子输送，但它提出了克服眼睛像差并在视网膜细胞膜上保持紧密聚焦点的重大技术挑战。此外，为了最大限度地减少附带损害并提高基因传递的吞吐量，我们向细胞引入了弱聚焦近红外（NIR）连续波（CW）或脉冲激光束，其中与细胞结合的金纳米棒局部增强了强度。细胞膜允许基因插入。通过改变曝光时间以及 CW NIR 光束的功率或脉冲 NIR 光束的能量，对纳米增强光学传输 (NOD) 进行参数优化。使用这种 NOD 方法，编码多特征视蛋白 (MCO) 的治疗基因被递送到离体和体内退化视网膜的空间目标区域。 NOD 介导的 MCO 在光感受器变性的目标视网膜区域中的细胞膜特异性表达将允许在环境光环境中功能恢复。
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