Gene editing technology and its recent progress in disease therapy
Received date: 2019-04-16
Revised date: 2019-05-29
Online published: 2019-07-02
Supported by
Supported by the National Natural Science Foundation of China(81670470);Supported by the National Natural Science Foundation of China(81873685)
Gene editing is a genetic manipulation technology which utilizes bacterial nucleases to accurately and efficiently modify DNA or RNA. Gene editing has broad applications in basic research, breeding, and drug screening, and it is gaining validity and applicability to the therapy of many diseases especially genetic-based disease. In this review, we summarize the development of gene editing technology, its different strategies and applications in the treatment of disease, and the research of gene editing therapy for genetic diseases (including base editor and epigenetic regulation) in the treatment of disorders and diseases of the blood system, liver, muscle and nervous system. Finally, we discuss the future development prospects of gene editing therapy.
Key words： gene editing; gene therapy; genetic diseases
Xuran Niu,Shuming Yin,Xi Chen,Tingting Shao,Dali Li . Gene editing technology and its recent progress in disease therapy[J]. Hereditas(Beijing), 2019 , 41(7) : 582 -598 . DOI: 10.16288/j.yczz.19-102
| [1] | Chu SY, Weng CY . Introduction to genetic/rare disease and the application of genetic counseling. Hu Li Za Zhi, 2017,64(5):11-17. | |||
| [1] | 褚思义, 翁纯英 . 遗传/罕见病简介及遗传咨询的应用. 护理杂志, 2017,64(5):11-17. | |||
| [2] | Darrow JJ . Luxturna: FDA documents reveal the value of a costly gene therapy. Drug Discov Today, 2019,24(4):949-954. | |||
| [3] | Han X, Ni W . Cost-Effectiveness analysis of glybera for the treatment of lipoprotein lipase deficiency. Value Health, 2015,18(7):A756. | |||
| [4] | Schimmer J, Breazzano S . Investor outlook: rising from the ashes; GSK's European approval of strimvelis for ADA-SCID. Hum Gene Ther Clin Dev, 2016,27(2):57-61. | |||
| [5] | Gupta SK, Shukla P . Gene editing for cell engineering: trends and applications. Crit Rev Biotechnol, 2017,37(5):672-684. | |||
| [6] | Takata M, Sasaki MS, Sonoda E, Morrison C, Hashimoto M, Utsumi H, Yamaguchi-Iwai Y, Shinohara A, Takeda S . Homologous recombination and non- homologous end-joining pathways of DNA double- strand break repair have overlapping roles in the maintenance of chromosomal integrity in vertebrate cells. EMBO J, 1998,17(18):5497-5508. | |||
| [7] | Lieber MR, Ma Y, Pannicke U, Schwarz K . Mechanism and regulation of human non-homologous DNA end- joining. Nat Rev Mol Cell Bio, 2003,4(9):712-720. | |||
| [8] | Joung JK, Sander JD . TALENs: a widely applicable technology for targeted genome editing. Nat Rev Mol Cell Bio, 2012,14(1):49-55. | |||
| [9] | Sander JD, Joung JK . CRISPR-Cas systems for editing, regulating and targeting genomes. Nat Biotechnol, 2014,32(4):347-355. | |||
| [10] | Komor AC, Badran AH, Liu DR . CRISPR-Based technologies for the manipulation of eukaryotic genomes. Cell, 2017,168(1-2):20-36. | |||
| [11] | Marcaida MJ, Prieto J, Redondo P . Crystal structure of I-DmoI in complex with its target DNA provides new insights into meganuclease engineering. Proc Natl Acad Sci USA, 2008,105(44):16888-16893. | |||
| [12] | Smith J, Grizot S, Arnould S, Duclert A, Epinat JC, Chames P, Prieto J, Redondo P, Blanco FJ, Bravo J, Montoya G, Paques F, Duchateau P . A combinatorial approach to create artificial homing endonucleases cleaving chosen sequences. Nucleic Acids Res, 2006,34(22):e149. | |||
| [13] | Silva G, Poirot L, Galetto R, Smith J, Montoya G, Duchateau P, Paques F . Meganucleases and other tools for targeted genome engineering: Perspectives and challenges for gene therapy. Curr Gene Ther, 2011,11(1):11-27. | |||
| [14] | Wang L, Smith J, Breton C, Clark P, Zhang J, Ying L, Che Y, Lape J, Bell P, Calcedo R, Buza EL, Saveliev A, Bartsevich
/
www.chinagene.cn
版权所有 © 中国科学院遗传与发育生物学研究所 中国遗传学会
地址：北京市朝阳区北辰西路1号院 中国科学院遗传与发育生物学研究所2号楼B-208 邮政编码：100101 联系电话：010-64807669 E-mail：yczz@genetics.ac.cn 
|