Gene therapy is expected to be utilized for the treatment of various diseases. However, the spatiotemporal resolution of current gene therapy technology is not high enough. In this study, we generated a new technology for spatiotemporally controllable gene therapy. We introduced optogenetic and CRISPR/Cas9 techniques into a recombinant adenovirus (Ad) vector, which is widely used in clinical trials and exhibits high gene transfer efficiency, to generate an illumination-dependent spatiotemporally controllable gene regulation system (designated the Opt/Cas-Ad system). We generated an Opt/Cas-Ad system that could regulate a potential tumor suppressor gene, and we examined the effectiveness of this system in cancer treatment using a xenograft tumor model. With the Opt/Cas-Ad system, highly selective tumor treatment could be performed by illuminating the tumor. In addition, Opt/Cas-Ad system-mediated tumor treatment could be stopped simply by turning off the light. We believe that our Opt/Cas-Ad system can enhance both the safety and effectiveness of gene therapy.

中文翻译：

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时空可控基因治疗的光遗传学修饰的腺病毒载体的产生。

基因疗法有望用于各种疾病的治疗。但是，当前基因治疗技术的时空分辨率还不够高。在这项研究中，我们产生了一种时空可控的基因治疗新技术。我们在重组腺病毒（Ad）载体中引入了光遗传学和CRISPR / Cas9技术，该载体在临床试验中被广泛使用并具有很高的基因转移效率，以产生光照依赖的时空可控基因调控系统（称为Opt / Cas-Ad系统）。我们生成了一个可以调节潜在肿瘤抑制基因的Opt / Cas-Ad系统，并使用异种移植肿瘤模型检查了该系统在癌症治疗中的有效性。使用Opt / Cas-Ad系统，可以通过照射肿瘤来进行高度选择性的肿瘤治疗。此外，仅需关闭灯，即可停止Opt / Cas-Ad系统介导的肿瘤治疗。我们相信，我们的Opt / Cas-Ad系统可以增强基因治疗的安全性和有效性。