CRISPR/Cas9 and TALE: beyond cut and paste

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• 作者：Liping Deng (1)
  Ruotong Ren (1)
  Jun Wu (2)
  Keiichiro Suzuki (2)
  Juan Carlos Izpisua Belmote (2)
  Guang-Hui Liu (1) (3) (4)
  
  1. National Laboratory of Biomacromolecules ; Institute of Biophysics ; Chinese Academy of Sciences ; Beijing ; 100101 ; China
  2. Gene Expression Laboratory ; Salk Institute for Biological Studies ; 10010 North Torrey Pines Road ; La Jolla ; CA ; 92037 ; USA
  3. Beijing Institute for Brain Disorders ; Beijing ; 100069 ; China
  4. Center for Molecular and Translational Medicine ; CMTM ; Beijing ; 100101 ; China
  
• 关键词：TALE ; CRISPR/Cas9 ; genome editing ; gene expression ; transcription regulation
• 刊名：Protein & Cell
• 出版年：2015
• 出版时间：March 2015
• 年：2015
• 卷：6
• 期：3
• 页码：157-159
• 全文大小：68 KB
• 参考文献：1. Chen, B (2013) Dynamic imaging of genomic loci in living human cells by an optimized CRISPR/Cas system. Cell 155: pp. 1479-1491 10.1016/j.cell.2013.12.001" target="_blank" title="It opens in new window">CrossRef
  2. Gaj, T, Gersbach, CA, Barbas, CF (2013) ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering. Trends Biotechnol 31: pp. 397-405 10.1016/j.tibtech.2013.04.004" target="_blank" title="It opens in new window">CrossRef
  3. Gilbert, LA (2013) CRISPR-mediated modular RNA-guided regulation of transcription in eukaryotes. Cell 154: pp. 442-451 10.1016/j.cell.2013.06.044" target="_blank" title="It opens in new window">CrossRef
  4. Hsu, PD (2013) DNA targeting specificity of RNA-guided Cas9 nucleases. Nat Biotechnol 31: pp. 827-832 10.1038/nbt.2647" target="_blank" title="It opens in new window">CrossRef
  5. Konermann, S (2013) Optical control of mammalian endogenous transcription and epigenetic states. Nature 500: pp. 472-476
  6. Konermann, S (2014) Genome-scale transcriptional activation by an engineered CRISPR-Cas9 complex. Nature 512: pp. 441-444 10.1038/nature13471" target="_blank" title="It opens in new window">CrossRef
  7. Li, M, Suzuki, K, Kim, NY, Liu, GH, Izpisua Belmonte, JC (2014) A cut above the rest: targeted genome editing technologies in human pluripotent stem cells. J Biol Chem 289: pp. 4594-4599 10.1074/jbc.R113.488247" target="_blank" title="It opens in new window">CrossRef
  8. Liu, GH (2014) Modelling Fanconi anemia pathogenesis and therapeutics using integration-free patient-derived iPSCs. Nat Commun 5: pp. 4330
  9. Maeder, ML (2013) Targeted DNA demethylation and activation of endogenous genes using programmable TALE-TET1 fusion proteins. Nat Biotechnol 31: pp. 1137-1142 10.1038/nbt.2726" target="_blank" title="It opens in new window">CrossRef
  10. Ma, H, Reyes-Gutierrez, P, Pederson, T (2013) Visualization of repetitive DNA sequences in human chromosomes with transcription activator-like effectors. Proc Natl Acad Sci USA 110: pp. 21048-21053 10.1073/pnas.1319097110" target="_blank" title="It opens in new window">CrossRef
  11. Mendenhall, EM (2013) Locus-specific editing of histone modifications at endogenous enhancers. Nat Biotechnol 31: pp. 1133-1136 10.1038/nbt.2701" target="_blank" title="It opens in new window">CrossRef
  12. Miyanari, Y, Ziegler-Birling, C, Torres-Padilla, ME (2013) Live visualization of chromatin dynamics with fluorescent TALEs. Nat Struct Mol Biol 20: pp. 1321-1324 10.1038/nsmb.2680" target="_blank" title="It opens in new window">CrossRef
  13. Pan, H, Zhang, W, Liu, GH (2011) Find and replace: editing human genome in pluripotent stem cells. Protein Cell 2: pp. 950-956 10.1007/s13238-011-1132-0" target="_blank" title="It opens in new window">CrossRef
  14. Smith, C (2014) Whole-genome sequencing analysis reveals high specificity of CRISPR/Cas9 and TALEN-based genome editing in human iPSCs. Cell Stem Cell 15: pp. 12-13 10.1016/j.stem.2014.06.011" target="_blank" title="It opens in new window">CrossRef
  15. Suzuki, K (2014) Targeted gene correction minimally impacts whole-genome mutational load in human-disease-specific induced pluripotent stem cell clones. Cell Stem Cell 15: pp. 31-36 10.1016/j.stem.2014.06.016" target="_blank" title="It opens in new window">CrossRef
  16. Therizols, P (2014) Chromatin decondensation is sufficient to alter nuclear organization in embryonic stem cells. Science 346: pp. 1238-1242 10.1126/science.1259587" target="_blank" title="It opens in new window">CrossRef
  17. Veres, A (2014) Low incidence of off-target mutations in individual CRISPR-Cas9 and TALEN targeted human stem cell clones detected by whole-genome sequencing. Cell Stem Cell 15: pp. 27-30 10.1016/j.stem.2014.04.020" target="_blank" title="It opens in new window">CrossRef
  18. Zalatan JG et al (2014) Engineering complex synthetic transcriptional programs with CRISPR RNA scaffolds. Cell 160:339鈥?50
  
• 刊物主题：Biochemistry, general; Protein Science; Cell Biology; Stem Cells; Human Genetics; Developmental Biology;
• 出版者：Springer Berlin Heidelberg
• ISSN：1674-8018

文摘

Nuclease-based genome editing has proven to be a powerful and promising tool for disease modeling and gene therapy. Recent advances in CRISPR/Cas and TALE indicate that they could also be used as a targeted regulator of gene expression, as well as being utilized for illuminating specific chromosomal structures or genomic regions.