Shanghai Institute for Advanced Immunochemical Studies, ShanghaiTech University, 99 Haike Road, Shanghai 201210, China.
Shanghai Institute for Advanced Immunochemical Studies, ShanghaiTech University, 99 Haike Road, Shanghai 201210, China.
J Control Release. 2016 Dec 28;244(Pt A):83-97. doi: 10.1016/j.jconrel.2016.11.014. Epub 2016 Nov 16.
The advent of site-specific nucleases, particularly CRISPR/Cas9, provides researchers with the unprecedented ability to manipulate genomic sequences. These nucleases are used to create model cell lines, engineer metabolic pathways, produce transgenic animals and plants, perform genome-wide functional screen and, most importantly, treat human diseases that are difficult to tackle by traditional medications. Considerable efforts have been devoted to improving the efficiency and specificity of nucleases for clinical applications. However, safe and efficient delivery methods remain the major obstacle for therapeutic gene editing. In this review, we summarize the recent progress on nuclease delivery methods, highlight their impact on the outcomes of gene editing and discuss the potential of different delivery approaches for therapeutic gene editing.
基因编辑技术是一种能够对基因组特定目标进行修饰的技术，包括定点突变、基因敲入、基因敲除等。该技术已经在基础研究和临床应用中得到了广泛的应用。通过基因编辑，我们可以对生物体的基因组进行精确的修饰，从而研究基因的功能、治疗疾病等。
近年来，基因编辑技术取得了飞速的发展，其中最引人注目的是 CRISPR/Cas9 系统。CRISPR/Cas9 系统是一种简单、高效、可编程的基因编辑工具，已经被广泛应用于基础研究和临床治疗中。
尽管基因编辑技术已经取得了很大的进展，但是仍然存在一些问题和挑战。例如，基因编辑的效率和特异性还有待提高，基因编辑可能会导致脱靶效应，从而引起不必要的副作用。此外，基因编辑技术还面临着伦理和法律等方面的挑战。
总之，基因编辑技术是一种非常有前途的生物技术，具有广泛的应用前景。随着技术的不断发展和完善，相信基因编辑技术将会在医学、农业、环境保护等领域发挥越来越重要的作用。
