Generation of Optogenetically Modified Adenovirus Vector for Spatiotemporally Controllable Gene Therapy,ACS Chemical Biology

当前位置： X-MOL 学术 › ACS Chem. Biol. › 论文详情

Our official English website, www.x-mol.net, welcomes your feedback! (Note: you will need to create a separate account there.)

Generation of Optogenetically Modified Adenovirus Vector for Spatiotemporally Controllable Gene Therapy
ACS Chemical Biology ( IF 3.5 ) Pub Date : 2018-01-12 00:00:00 , DOI: 10.1021/acschembio.7b01058
Kazuo Takayama _{1,

2,

3} , Hiroyuki Mizuguchi _{1,

3,

4}

Affiliation

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：

用于时空可控基因治疗的光遗传学修饰腺病毒载体的产生

基因疗法有望用于治疗各种疾病。然而，目前的基因治疗技术的时空分辨率还不够高。在这项研究中，我们开发了一种时空可控基因治疗新技术。我们将光遗传学和CRISPR/Cas9技术引入广泛应用于临床试验并表现出高基因转移效率的重组腺病毒（Ad）载体中，生成依赖于光照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。利用Opt/Cas-Ad系统，可以通过照射肿瘤来进行高选择性的肿瘤治疗。此外，只需关灯即可停止 Opt/Cas-Ad 系统介导的肿瘤治疗。我们相信我们的 Opt/Cas-Ad 系统可以提高基因治疗的安全性和有效性。

更新日期：2018-01-12

点击分享查看原文

点击收藏

阅读更多本刊最新论文本刊介绍/投稿指南11