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近红外光可激活的化学诱导CRISPR系统

Near-infrared light activatable chemically induced CRISPR system.

作者信息

Zhang Lei, Zhang Xuejun, Qiu Le, Mao Song, Sheng Jia, Chen Liming, Khan Umar, Upputuri Paul K, Zakharov Yuri N, Coughlan Mark F, Perelman Lev T

机构信息

Center for Advanced Biomedical Imaging and Photonics, Division of Gastroenterology, Department of Medicine, Beth Israel Deaconess Medical Center, Harvard University, Boston, MA, USA.

The RNA Institute, Department of Chemistry, University at Albany, Albany, NY, USA.

出版信息

Light Sci Appl. 2025 Jul 1;14(1):229. doi: 10.1038/s41377-025-01917-8.

Abstract

The biggest challenge in using CRISPR technologies, which limits their widespread application in medicine, is off-target effects. These effects could, in principle, be minimized by ensuring that CRISPR is activated primarily in the targeted cells, thereby reducing the likelihood of unintended genetic modifications in non-target tissues. Therefore, the development of a light activatable CRISPR approach to dynamically control gene activation in both space and time would be highly beneficial. A drawback is that the overwhelming majority of recently introduced light activatable CRISPR systems require UV or blue light exposure, severely limiting the penetration depth of light in tissue at which CRISPR can be activated, and, in the case of UV light, raising safety concerns. A small number of systems that activate CRISPR using longer wavelengths are hindered by either slow light activation or issues related to toxicity and biocompatibility of the proposed techniques in humans. To address this, we developed a split-Cas9/dCas9 system in which activation is achieved through a near-infrared photocleavable dimerization complex. This photoactivation method can be safely used in humans in vivo, easily adapted to different split-Cas9/dCas9 systems, and enables rapid, spatially precise light activation across various cell types.

摘要

使用CRISPR技术的最大挑战是脱靶效应,这限制了它们在医学上的广泛应用。原则上,通过确保CRISPR主要在靶细胞中被激活,从而降低非靶组织中意外基因修饰的可能性,可以将这些效应最小化。因此,开发一种光激活的CRISPR方法来在空间和时间上动态控制基因激活将非常有益。一个缺点是,绝大多数最近引入的光激活CRISPR系统需要暴露于紫外线或蓝光下,这严重限制了CRISPR可被激活的光在组织中的穿透深度,并且在紫外线的情况下,还引发了安全问题。少数使用较长波长激活CRISPR的系统受到光激活缓慢或与所提出技术在人体中的毒性和生物相容性相关问题的阻碍。为了解决这个问题,我们开发了一种分裂Cas9/dCas9系统,其中激活是通过近红外光可裂解二聚化复合物实现的。这种光激活方法可以在人体体内安全使用,很容易适应不同的分裂Cas9/dCas9系统,并能够在各种细胞类型中实现快速、空间精确的光激活。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ebc6/12214668/2d94fe01a56e/41377_2025_1917_Fig1_HTML.jpg

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