Efficient bi-allelic gene knockout and site-specific knock-in mediated by TALENs in pigs

Scientific Reports

Volumn 4, Issue , 2014, Pages

  Yao, Jing ^a   Huang, Jiaojiao ^a   Hai, Tang ^a   Wang, Xianlong ^a   Qin, Guosong ^a   Zhang, Hongyong ^a   Wu, Rong ^a   Cao, Chunwei ^a   Xi, Jianzhong Jeff ^b   Yuan, Zengqiang ^a   Zhao, Jianguo ^a  

^a INSTITUTE OF GEOLOGY AND GEOPHYSICS   (China)

^b PEKING UNIVERSITY   (China)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA-1,3-GALACTOSYLTRANSFERASE 1, PORCINE; GALACTOSYLTRANSFERASE; HYBRID PROTEIN; PARKIN; RESTRICTION ENDONUCLEASE; UBIQUITIN PROTEIN LIGASE;

ALLELE; ANIMAL; CELL CULTURE; CELL NUCLEUS TRANSPLANTATION; CLONING; FEMALE; GENE EXPRESSION REGULATION; GENE INACTIVATION; GENE TARGETING; GENETIC ENGINEERING; GENETICS; MALE; MOLECULAR GENETICS; NUCLEOTIDE SEQUENCE; PIG;

ALLELES; ANIMALS; BASE SEQUENCE; CELLS, CULTURED; CLONING, ORGANISM; DNA RESTRICTION ENZYMES; FEMALE; GALACTOSYLTRANSFERASES; GENE KNOCK-IN TECHNIQUES; GENE KNOCKOUT TECHNIQUES; GENETIC ENGINEERING; MALE; MOLECULAR SEQUENCE DATA; MUTAGENESIS, INSERTIONAL; NUCLEAR TRANSFER TECHNIQUES; RECOMBINANT FUSION PROTEINS; SUS SCROFA; UBIQUITIN-PROTEIN LIGASES;

EID: 84922634221     PISSN: None     EISSN: 20452322     Source Type: Journal    
DOI: 10.1038/srep06926     Document Type: Article

References (45)

1. Prather, R. S., Shen, M. & Dai, Y. Genetically modified pigs for medicine and agriculture. Biotechnol Genet Eng Rev 25, 245-265 (2008).
2. Carter, D. B. et al. Phenotyping of transgenic cloned piglets. Cloning Stem Cells 4, 131-145 (2002).
3. Rogers, C. S. et al. Disruption of the CFTR gene produces amodel of cystic fibrosis in newborn pigs. Science 321, 1837-1841 (2008).
4. Aigner, B. et al. Transgenic pigs as models for translational biomedical research. J Mol Med (Berl) 88, 653-664 (2010).
5. Yang, D. et al. Expression of Huntington's disease protein results in apoptotic neurons in the brains of cloned transgenic pigs. Hum Mol Genet 19, 3983-3994 (2010).
6. Carlson, D. F. et al. Efficient TALEN-mediated gene knockout in livestock. Proc Natl Acad Sci U S A 109, 17382-17387 (2012).
7. Bibikova, M., Beumer, K., Trautman, J. K. & Carroll, D. Enhancing gene targeting with designed zinc finger nucleases. Science 300, 764 (2003).
8. Whyte, J. J. et al. Gene targeting with zinc finger nucleases to produce cloned eGFP knockout pigs. Mol Reprod Dev 78, 2 (2011).
9. Yang, D. et al. Generation of PPARgamma mono-allelic knockout pigs via zincfinger nucleases and nuclear transfer cloning. Cell Res 21, 979-982 (2011).
10. Hauschild, J. et al. Efficient generation of a biallelic knockout in pigs using zincfinger nucleases. Proc Natl Acad Sci U S A 108, 12013-12017 (2011).
11. Hockemeyer, D. et al. Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases. Nat Biotechnol 27, 851-857 (2009).
12. Geurts, A. M. et al. Knockout rats via embryo microinjection of zinc-finger nucleases. Science 325, 433 (2009).
13. Cornu, T. I. et al. DNA-binding specificity is a major determinant of the activity and toxicity of zinc-finger nucleases. Mol Ther 16, 352-358 (2008).
14. Radecke, S., Radecke, F., Cathomen, T. & Schwarz, K. Zinc-finger nucleaseinduced gene repair with oligodeoxynucleotides: wanted and unwanted target locus modifications. Mol Ther 18, 743-753 (2010).
15. Carlson, D. F., Fahrenkrug, S. C. & Hackett, P. B. Targeting DNA With Fingers and TALENs. Mol Ther Nucleic Acids 1, e3 (2012).
16. Christian, M. et al. Targeting DNA double-strand breaks with TAL effector nucleases. Genetics 186, 757-761 (2010).
17. Sander, J. D. et al. Targeted gene disruption in somatic zebrafish cells using engineered TALENs. Nat Biotechnol 29, 697-698 (2011).
18. Bedell, V. M. et al. In vivo genome editing using a high-efficiency TALEN system. Nature 491, 114-118 (2012).
19. Tesson, L. et al. Knockout rats generated by embryo microinjection of TALENs. Nat Biotechnol 29, 695-696 (2011).
20. Thomas, B. & Beal, M. F. Molecular insights into Parkinson's disease. F1000Med Rep 3, 7 (2011).
21. Bonifati, V. et al. Mutations in the DJ-1 gene associated with autosomal recessive early-onset parkinsonism. Science 299, 256-259 (2003).
22. Goldberg, M. S. et al. Parkin-deficient mice exhibit nigrostriatal deficits but not loss of dopaminergic neurons. J Biol Chem 278, 43628-43635 (2003).
23. Itier, J. M. et al. Parkin gene inactivation alters behaviour and dopamine neurotransmission in the mouse. Hum Mol Genet 12, 2277-2291 (2003).
24. Von Coelln, R. et al. Loss of locus coeruleus neurons and reduced startle in parkin null mice. Proc Natl Acad Sci U S A 101, 10744-10749 (2004).
25. Chen, L. et al. Age-dependent motor deficits and dopaminergic dysfunction in DJ-1 null mice. J Biol Chem 280, 21418-21426 (2005).
26. Perez, F. A. & Palmiter, R. D. Parkin-deficient mice are not a robust model of parkinsonism. Proc Natl Acad Sci U S A 102, 2174-2179 (2005).
27. Rodriguez-Navarro, J. A. et al. Mortality, oxidative stress and tau accumulation during ageing in parkin null mice. J Neurochem 103, 98-114 (2007).
28. Andres-Mateos, E. et al. DJ-1 gene deletion reveals that DJ-1 is an atypical peroxiredoxin-like peroxidase. Proc Natl Acad Sci U S A 104, 14807-14812 (2007).
29. Rousseaux, M. W. et al. Progressive dopaminergic cell loss with unilateral-tobilateral progression in a genetic model of Parkinson disease. Proc Natl Acad Sci U S A 109, 15918-15923 (2012).
30. Lai, L. et al. Production of alpha-1, 3-galactosyltransferase knockout pigs by nuclear transfer cloning. Science 295, 1089-1092 (2002).
31. Lei, Y. et al. Efficient targeted gene disruption in Xenopus embryos using engineered transcription activator-like effector nucleases (TALENs). Proc Natl Acad Sci U S A 109, 17484-17489 (2012).
32. Xin, J. et al. Highly efficient generation of GGTA1 biallelic knockout inbred minipigs with TALENs. PLoS One 8, e84250 (2013).
33. Grau, J., Boch, J. & Posch, S. TALENoffer: genome-wide TALEN off-target prediction. Bioinformatics 29, 2931-2932 (2013).
34. Chen, F. et al. High-frequency genome editing using ssDNA oligonucleotides with zinc-finger nucleases. Nat Methods 8, 753-755 (2011).
35. Sung, Y. H. et al. Knockout mice created by TALEN-mediated gene targeting. Nat Biotechnol 31, 23-24 (2013).
36. Fu, Y. et al. High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells. Nat Biotechnol 31, 822-826 (2013).
37. Tan, W. et al. Efficient nonmeiotic allele introgression in livestock using custom endonucleases. Proc Natl Acad Sci U S A 110, 16526-16531 (2013).
38. Soldner, F. et al. Generation of isogenic pluripotent stem cells differing exclusively at two early onset Parkinson point mutations. Cell 146, 318-331 (2011).
39. Meyer, M., Ortiz, O., Hrabe De Angelis, M., Wurst, W. & Kuhn, R. Modeling disease mutations by gene targeting in one-cell mouse embryos. Proc Natl Acad Sci U S A 109, 9354-9359 (2012).
40. Wefers, B. et al. Direct production of mouse disease models by embryo microinjection of TALENs and oligodeoxynucleotides. Proc Natl Acad Sci U S A 110, 3782-3787 (2013).
41. Wang, H. et al. One-step generation of mice carrying mutations in multiple genes by CRISPR/Cas-mediated genome engineering. Cell 153, 910-918 (2013).
42. Ross, J. W. et al. Optimization of square-wave electroporation for transfection of porcine fetal fibroblasts. Transgenic Res 19, 611-620 (2010).
43. Gospodarowicz, D., Ferrara, N., Schweigerer, L. & Neufeld, G. Structural characterization and biological functions of fibroblast growth factor. Endocr Rev 8, 95-114 (1987).
44. Rifkin, D. B. & Moscatelli, D. Recent developments in the cell biology of basic fibroblast growth factor. J Cell Biol 109, 1-6 (1989).
45. Miller, J. C. et al. A TALE nuclease architecture for efficient genome editing. Nat Biotechnol 29, 143-148 (2011).