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Generation of Optogenetically Modified Adenovirus Vector for Spatiotemporally Controllable Gene Therapy.

Abstract：

: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.

journal_name

ACS Chem Biol

journal_title

ACS chemical biology

authors

Takayama K,Mizuguchi H

doi

10.1021/acschembio.7b01058

subject

Has Abstract

pub_date

2018-02-16 00:00:00

pages

449-454

issue

2

eissn

1554-8929

issn

1554-8937

journal_volume

13

pub_type

杂志文章

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