Jo Young-Il, Suresh Bharathi, Kim Hyongbum, Ramakrishna Suresh
Brandeis University, Waltham, MA, United States.
Department of Pharmacology, Brain Korea 21 PLUS Project for Medical Science, Yonsei University College of Medicine, Seoul, South Korea; Department of Physiology, Graduate Program of Nano Science and Technology, Yonsei University, Seoul, South Korea.
Biochim Biophys Acta. 2015 Dec;1856(2):234-43. doi: 10.1016/j.bbcan.2015.09.003. Epub 2015 Nov 11.
While human gene therapy has gained significant attention for its therapeutic promise, CRISPR/Cas9 technology has made a breakthrough as an efficient genome editing tool by emulating prokaryotic immune defense mechanisms. Although many studies have found that CRISPR/Cas9 technology is more efficient, specific and manipulable than previous generations of gene editing tools, it can be further improved by elevating its overall efficiency in a higher frequency of genome modifications and reducing its off-target effects. Here, we review the development of CRISPR/Cas9 technology, focusing on enhancement of its sequence specificity, reduction of off-target effects and delivery systems. Moreover, we describe recent successful applications of CRISPR/Cas9 technology in laboratory and clinical studies.
尽管人类基因治疗因其潜在的治疗前景而备受关注,但CRISPR/Cas9技术通过模拟原核生物免疫防御机制,作为一种高效的基因组编辑工具取得了突破。尽管许多研究发现CRISPR/Cas9技术比前几代基因编辑工具更高效、更具特异性且更易于操作,但仍可通过提高其在更高频率基因组修饰中的整体效率以及减少脱靶效应来进一步改进。在此,我们综述了CRISPR/Cas9技术的发展,重点关注其序列特异性的增强、脱靶效应的降低以及递送系统。此外,我们还描述了CRISPR/Cas9技术最近在实验室和临床研究中的成功应用。