通过CRISPR/Cas9介导的同源性非依赖靶向整合进行体内基因组编辑。
In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration.
作者信息
Suzuki Keiichiro, Tsunekawa Yuji, Hernandez-Benitez Reyna, Wu Jun, Zhu Jie, Kim Euiseok J, Hatanaka Fumiyuki, Yamamoto Mako, Araoka Toshikazu, Li Zhe, Kurita Masakazu, Hishida Tomoaki, Li Mo, Aizawa Emi, Guo Shicheng, Chen Song, Goebl April, Soligalla Rupa Devi, Qu Jing, Jiang Tingshuai, Fu Xin, Jafari Maryam, Esteban Concepcion Rodriguez, Berggren W Travis, Lajara Jeronimo, Nuñez-Delicado Estrella, Guillen Pedro, Campistol Josep M, Matsuzaki Fumio, Liu Guang-Hui, Magistretti Pierre, Zhang Kun, Callaway Edward M, Zhang Kang, Belmonte Juan Carlos Izpisua
机构信息
Gene Expression Laboratory, Salk Institute for Biological Studies, 10010 N. Torrey Pines Rd, La Jolla, California 92037, USA.
Laboratory for Cell Asymmetry, RIKEN Center for Developmental Biology, 2-2-3 Minatojima-Minamimachi, Chuo-ku, Kobe 650-0047, Japan.
出版信息
Nature. 2016 Dec 1;540(7631):144-149. doi: 10.1038/nature20565. Epub 2016 Nov 16.
Targeted genome editing via engineered nucleases is an exciting area of biomedical research and holds potential for clinical applications. Despite rapid advances in the field, in vivo targeted transgene integration is still infeasible because current tools are inefficient, especially for non-dividing cells, which compose most adult tissues. This poses a barrier for uncovering fundamental biological principles and developing treatments for a broad range of genetic disorders. Based on clustered regularly interspaced short palindromic repeat/Cas9 (CRISPR/Cas9) technology, here we devise a homology-independent targeted integration (HITI) strategy, which allows for robust DNA knock-in in both dividing and non-dividing cells in vitro and, more importantly, in vivo (for example, in neurons of postnatal mammals). As a proof of concept of its therapeutic potential, we demonstrate the efficacy of HITI in improving visual function using a rat model of the retinal degeneration condition retinitis pigmentosa. The HITI method presented here establishes new avenues for basic research and targeted gene therapies.
通过工程核酸酶进行靶向基因组编辑是生物医学研究中一个令人兴奋的领域,具有临床应用潜力。尽管该领域取得了快速进展,但体内靶向转基因整合仍然不可行,因为目前的工具效率低下,尤其是对于构成大多数成年组织的非分裂细胞。这为揭示基本生物学原理和开发针对广泛遗传疾病的治疗方法带来了障碍。基于成簇规律间隔短回文重复序列/Cas9(CRISPR/Cas9)技术,我们在此设计了一种不依赖同源性的靶向整合(HITI)策略,该策略能够在体外的分裂细胞和非分裂细胞中,更重要的是在体内(例如,在出生后哺乳动物的神经元中)实现强大的DNA敲入。作为其治疗潜力概念验证,我们使用视网膜色素变性视网膜退化疾病的大鼠模型证明了HITI在改善视觉功能方面的功效。本文提出的HITI方法为基础研究和靶向基因治疗开辟了新途径。