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

被引:329
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
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 93 条
  • [1] Highly active zinc-finger nucleases by extended modular assembly
    Bhakta, Mital S.
    Henry, Isabelle M.
    Ousterout, David G.
    Das, Kumitaa Theva
    Lockwood, Sarah H.
    Meckler, Joshua F.
    Wallen, Mark C.
    Zykovich, Artem
    Yu, Yawei
    Leo, Heather
    Xu, Lifeng
    Gersbach, Charles A.
    Segal, David J.
    [J]. GENOME RESEARCH, 2013, 23 (03) : 530 - 538
  • [2] Breaking the Code of DNA Binding Specificity of TAL-Type III Effectors
    Boch, Jens
    Scholze, Heidi
    Schornack, Sebastian
    Landgraf, Angelika
    Hahn, Simone
    Kay, Sabine
    Lahaye, Thomas
    Nickstadt, Anja
    Bonas, Ulla
    [J]. SCIENCE, 2009, 326 (5959) : 1509 - 1512
  • [3] TAL Effectors: Customizable Proteins for DNA Targeting
    Bogdanove, Adam J.
    Voytas, Daniel F.
    [J]. SCIENCE, 2011, 333 (6051) : 1843 - 1846
  • [4] Targeted Genome Modification in Mice Using Zinc-Finger Nucleases
    Carbery, Iara D.
    Ji, Diana
    Harrington, Anne
    Brown, Victoria
    Weinstein, Edward J.
    Liaw, Lucy
    Cui, Xiaoxia
    [J]. GENETICS, 2010, 186 (02) : 451 - U37
  • [5] Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting
    Cermak, Tomas
    Doyle, Erin L.
    Christian, Michelle
    Wang, Li
    Zhang, Yong
    Schmidt, Clarice
    Baller, Joshua A.
    Somia, Nikunj V.
    Bogdanove, Adam J.
    Voytas, Daniel F.
    [J]. NUCLEIC ACIDS RESEARCH, 2011, 39 (12) : e82
  • [6] High-frequency genome editing using ssDNA oligonucleotides with zinc-finger nucleases
    Chen, Fuqiang
    Pruett-Miller, Shondra M.
    Huang, Yuping
    Gjoka, Monika
    Duda, Katarzyna
    Taunton, Jack
    Collingwood, Trevor N.
    Frodin, Morten
    Davis, Gregory D.
    [J]. NATURE METHODS, 2011, 8 (09) : 753 - U96
  • [7] Analysis of off-target effects of CRISPR/Cas-derived RNA-guided endonucleases and nickases
    Cho, Seung Woo
    Kim, Sojung
    Kim, Yongsub
    Kweon, Jiyeon
    Kim, Heon Seok
    Bae, Sangsu
    Kim, Jin-Soo
    [J]. GENOME RESEARCH, 2014, 24 (01) : 132 - 141
  • [8] Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease
    Cho, Seung Woo
    Kim, Sojung
    Kim, Jong Min
    Kim, Jin-Soo
    [J]. NATURE BIOTECHNOLOGY, 2013, 31 (03) : 230 - 232
  • [9] Targeting DNA Double-Strand Breaks with TAL Effector Nucleases
    Christian, Michelle
    Cermak, Tomas
    Doyle, Erin L.
    Schmidt, Clarice
    Zhang, Feng
    Hummel, Aaron
    Bogdanove, Adam J.
    Voytas, Daniel F.
    [J]. GENETICS, 2010, 186 (02) : 757 - U476
  • [10] Multiplex Genome Engineering Using CRISPR/Cas Systems
    Cong, Le
    Ran, F. Ann
    Cox, David
    Lin, Shuailiang
    Barretto, Robert
    Habib, Naomi
    Hsu, Patrick D.
    Wu, Xuebing
    Jiang, Wenyan
    Marraffini, Luciano A.
    Zhang, Feng
    [J]. SCIENCE, 2013, 339 (6121) : 819 - 823