基因编辑技术及其在基因治疗中的应用

doi:10.16288/j.yczz.18-142

[an error occurred while processing this directive]

Hereditas(Beijing) ›› 2019, Vol. 41 ›› Issue (1): 18-27.doi: 10.16288/j.yczz.18-142

Previous Articles Next Articles

Research advance and application in the gene therapy of gene editing technologies

Yunxiao Ren^1,^2,³,Rudan Xiao^1,⁴,Xiaomin Lou^1,^2,^3,⁴,Xiangdong Fang^1,^2,^3,⁴

^1. CAS Key Laboratory of Genome Sciences and Information, Beijing Institute of Genomics, Chinese Academy of Sciences, Beijing 100101, China
^2. Sino-Danish College, University of Chinese Academy of Sciences, Bejing 101408, China
^3. Sino-Danish Center for Education and Research, Beijing 101408, China
^4. Life Sciences College, University of Chinese Academy of Sciences, Beijing 100049, China

Received:2018-08-16 Revised:2018-10-27 Online:2019-01-20 Published:2018-12-06
Supported by:
[Supported by the National Natural Science Foundation of China (Nos. 31471115, 81670109, 81672698)]

Abstract

Abstract:

Gene editing technologies are used to specifically edit the target sequence. With the development of zinc finger nuclease (ZFN), transcription activator-like effector nuclease (TALEN), regular clustering of short palindrome repeats (CRISPR) and single base editing (BE) techniques, gene editing technologies not only provide powerful tools for gene functional studies, but also offer new therapeutic strategies in biomedical research. Gene editing has demonstrated broad application prospects in the gene therapy field, as well as in the construction of animal and cell models, drug target screening and gene functional research. In this review, we summarize several typical gene editing technologies, their characteristics and applications in gene therapy and discusses their opportunities and challenges in gene therapy, thereby providing critical insights and references on the clinical application of gene editing technologies.

Key words: gene editing technologies, CRISPR/Cas9, BE, gene therapy

Yunxiao Ren, Rudan Xiao, Xiaomin Lou, Xiangdong Fang. Research advance and application in the gene therapy of gene editing technologies[J]. Hereditas(Beijing), 2019, 41(1): 18-27.

References

[1]	Mali P, Yang L, Esvelt KM, Aach J, Guell M, DiCarlo JE, Norville JE, Church GM . RNA-guided human genome engineering via Cas9 prashant. Science, 2013,339(6121):823-826. [DOI]
[2]	Hsu PD, Lander ES, Zhang F . Development and applications of CRISPR-Cas9 for genome engineering. Cell, 2014,157(6):1262-1278. [DOI]
[3]	Kim H, Kim JS . A guide to genome engineering with programmable nucleases. Nat Rev Genet, 2014,15(5):321-334. [DOI]
[4]	Gaj T, Gersbach CA, Barbas CF. ZFN , TALEN,CRISPR/Cas-based methods for genome engineering. Trends Biotechnol, 2013,31(7):397-405. [DOI]
[5]	Ahmad HI, Ahmad MJ, Asif AR, Adnan M, Iqbal MK, Mehmood K, Muhammad SA, Bhuiyan AA, Elokil A, Du X, Zhao C, Liu X, Xie S . A review of crispr-based genome editing: Survival, evolution and challenges. Curr Issues Mol Biol, 2018,28:47-68. [DOI]
[6]	Musunuru K . The hope and hype of CRISPR-Cas9 genome editing: A review. JAMA Cardiol, 2017,2(8):914-919. [DOI]
[7]	Li L, Wu LP, Chandrasegaran S . Functional domains in Fok I restriction endonuclease. Proc Natl Acad Sci USA, 1992,89(10):4275-4279. [DOI]
[8]	Kim YG, Chandrasegaran S . Chimeric restriction endonuclease. Proc Natl Acad Sci USA, 1994,91(3):883-887. [DOI]
[9]	Kim YG, Cha J, Chandrasegaran S . Hybrid restriction enzymes: zinc finger fusions to Fok I cleavage domain. Proc Natl Acad Sci USA, 1996,93(3):1156-1160. [DOI]
[10]	Durai S, Mani M, Kandavelou K, Wu J, Porteus MH, Chandrasegaran S . Zinc finger nucleases: Custom- designed molecular scissors for genome engineering of plant and mammalian cells. Nucleic Acids Res, 2005,33(18):5978-5990. [DOI]
[11]	Xiao A, Hu YY, Wang WY, Yang ZP, Wang ZX, Huang P, Tong XJ, Zhang B, Lin S . Progress in zinc finger nuclease engineering for targeted genome modification. Hereditas (Beijing), 2011,33(7):665-683.
[11]	肖安, 胡莹莹, 王唯晔, 杨志芃, 王展翔, 黄鹏, 佟向军, 张博, 林硕 . 人工锌指核酸酶介导的基因组定点修饰技术. 遗传, 2011,33(7):665-683. [DOI]
[12]	Bibikova M, Golic M, Golic KG, Carroll D . Targeted chromosomal cleavage and mutagenesis in Drosophila using zinc-finger nucleases. Genetics, 2002,161(3):1169-1175. [DOI]
[13]	Bibikova M, Beumer K, Trautman JK, Carroll D . Enhancing gene targeting with designed zinc finger nucleases. Science, 2003,300(5620):764. [DOI]
[14]	Kim J-S . Genome editing comes of age. Nat Protoc, 2016,11(9):1573-1578. [DOI]
[15]	Hatada I, Horii T . Genome editing: A breakthrough in life science and medicine. Endocr J, 2016,63(2):105-110. [DOI]

Metrics

Comments

www.chinagene.cn
备案号：京ICP备09063187号

Research advance and application in the gene therapy of gene editing technologies

RichHTML

PDF (PC)

Knowledge

Abstract

Cite this article

share this article

References

Related Articles 15

Recommended Articles

Metrics

Comments