Nishio Maui, Yamagishi Ayana, Tsukakoshi Kaori, Kato Yoshio, Nakamura Chikashi, Ikebukuro Kazunori
Department of Biotechnology and Life Science, Tokyo University of Agriculture and Technology, Tokyo, Japan.
Biomedical Research Institute, National Institute of Advanced Industrial Science and Technology (AIST), Ibaraki, Japan.
Methods Mol Biol. 2018;1867:165-174. doi: 10.1007/978-1-4939-8799-3_12.
Genome editing with site-specific nucleases (SSNs) may be effective for gene therapy, as SSNs can modify target genes. However, the main limitation of genome editing for clinical use is off-target effects by excess amounts of SSNs within cells. Therefore, a controlled delivery system for SSNs is necessary. Previously we have reported on a zinc finger nuclease (ZFN) delivery system, which combined DNA aptamers against FokI nuclease domain (FokI) and nanoneedles. Here, we describe how DNA aptamers against FokI were selected and characterized for genome editing applications.
使用位点特异性核酸酶（SSN）进行基因组编辑可能对基因治疗有效，因为SSN可以修饰靶基因。然而，基因组编辑用于临床的主要限制是细胞内过量的SSN产生的脱靶效应。因此，需要一种用于SSN的可控递送系统。此前我们报道了一种锌指核酸酶（ZFN）递送系统，该系统将针对FokI核酸酶结构域（FokI）的DNA适体与纳米针相结合。在这里，我们描述了如何选择针对FokI的DNA适体并对其进行表征以用于基因组编辑应用。
