Expanding the genetic editing tool kit: ZFNs, TALENs, and CRISPR-Cas9

被引:308
|
作者
Gupta, Rajat M.
Musunuru, Kiran
机构
[1] Harvard Univ, Dept Stem Cell & Regenerat Biol, Cambridge, MA 02138 USA
[2] Brigham & Womens Hosp, Div Cardiovasc Med, Boston, MA 02115 USA
来源
JOURNAL OF CLINICAL INVESTIGATION | 2014年 / 124卷 / 10期
关键词
ZINC-FINGER NUCLEASES; ONE-STEP GENERATION; HUMAN-CELLS; HOMOLOGOUS RECOMBINATION; EMBRYO MICROINJECTION; KNOCKOUT RATS; GENOME MODIFICATION; EFFECTOR NUCLEASES; BETA-GLOBIN; HUMAN IPSCS;
D O I
10.1172/JCI72992
中图分类号
R-3 [医学研究方法]; R3 [基础医学];
学科分类号
1001 ;
摘要
The past decade has been one of rapid innovation in genome-editing technology. The opportunity now exists for investigators to manipulate virtually any gene in a diverse range of cell types and organisms with targeted nucleases designed with sequence-specific DNA-binding domains. The rapid development of the field has allowed for highly efficient, precise, and now cost-effective means by which to generate human and animal models of disease using these technologies. This review will outline the recent development of genome-editing technology, culminating with the use of CRISPR-Cas9 to generate novel mammalian models of disease. While the road to using this same technology for treatment of human disease is long, the pace of innovation over the past five years and early successes in model systems build-anticipation for this prospect.
引用
收藏
页码:4154 / 4161
页数:8
相关论文
共 50 条
  • [1] Delivery and therapeutic applications of gene editing technologies ZFNs, TALENs, and CRISPR/Cas9
    LaFountaine, Justin S.
    Fathe, Kristin
    Smyth, Hugh D. C.
    [J]. INTERNATIONAL JOURNAL OF PHARMACEUTICS, 2015, 494 (01) : 180 - 194
  • [2] Expanding the genome-editing range of CRISPR-Cas9 in rice
    Xu, Xiuling
    [J]. NATIONAL SCIENCE REVIEW, 2018, 5 (01) : 6 - 6
  • [3] Genome centric engineering using ZFNs, TALENs and CRISPR-Cas9 systems for trait improvement and disease control in Animals
    Wani, Atif Khurshid
    Akhtar, Nahid
    Singh, Reena
    Prakash, Ajit
    Raza, Sayed Haidar Abbas
    Cavalu, Simona
    Chopra, Chirag
    Madkour, Mahmoud
    Elolimy, Ahmed
    Hashem, Nesrein M.
    [J]. VETERINARY RESEARCH COMMUNICATIONS, 2023, 47 (01) : 1 - 16
  • [4] Genome centric engineering using ZFNs, TALENs and CRISPR-Cas9 systems for trait improvement and disease control in Animals
    Atif Khurshid Wani
    Nahid Akhtar
    Reena Singh
    Ajit Prakash
    Sayed Haidar Abbas Raza
    Simona Cavalu
    Chirag Chopra
    Mahmoud Madkour
    Ahmed Elolimy
    Nesrein M. Hashem
    [J]. Veterinary Research Communications, 2023, 47 : 1 - 16
  • [5] The application of CRISPR-Cas9 genome editing tool in cancer immunotherapy
    Wu, Hong-yan
    Cao, Chun-yu
    [J]. BRIEFINGS IN FUNCTIONAL GENOMICS, 2019, 18 (02) : 129 - 132
  • [6] CRISPR-Cas9; an efficient tool for precise plant genome editing
    Islam, Waqar
    [J]. MOLECULAR AND CELLULAR PROBES, 2018, 39 : 47 - 52
  • [7] CRISPR-Cas9 Genome Editing Tool for the Production of Industrial Biopharmaceuticals
    Amjad Hayat Khan
    Gee Jun Tye
    Rahmah Noordin
    [J]. Molecular Biotechnology, 2020, 62 : 401 - 411
  • [8] CRISPR-Cas9 Genome Editing Tool for the Production of Industrial Biopharmaceuticals
    Khan, Amjad Hayat
    Tye, Gee Jun
    Noordin, Rahmah
    [J]. MOLECULAR BIOTECHNOLOGY, 2020, 62 (09) : 401 - 411
  • [9] CRISPR-Cas9: Tool for Qualitative and Quantitative Plant Genome Editing
    Noman, Ali
    Aqeel, Muhammad
    He, Shuilin
    [J]. FRONTIERS IN PLANT SCIENCE, 2016, 7
  • [10] CRISPR-Cas9 genome editing in iPSCs for functional genetic screening
    Isachenko, Nadja
    Hu, Dongfang
    Chenchik, Alex
    Diehl, Paul
    Tedesco, Donato
    [J]. MOLECULAR CANCER THERAPEUTICS, 2023, 22 (12)