Generation of hypoxanthine phosphoribosyltransferase gene knockout rabbits by homologous recombination and gene trapping through somatic cell nuclear transfer

Scientific Reports

Volumn 5, Issue , 2015, Pages

  Yin, Mingru ^a   Jiang, Weihua ^a   Fang, Zhenfu ^a   Kong, Pengcheng ^a   Xing, Fengying ^a   Li, Yao ^a   Chen, Xuejin ^a   Li, Shangang ^a  

^a SHANGHAI JIAO TONG UNIVERSITY SCHOOL OF MEDICINE   (China)

Author keywords

[No Author keywords available]

Indexed keywords

HYPOXANTHINE PHOSPHORIBOSYLTRANSFERASE;

ANIMAL; CELL NUCLEUS TRANSPLANTATION; FEMALE; GENE INACTIVATION; GENETICS; HOMOLOGOUS RECOMBINATION; MALE; PROCEDURES; RABBIT; TRANSGENIC ANIMAL;

ANIMALS; ANIMALS, GENETICALLY MODIFIED; FEMALE; GENE KNOCKOUT TECHNIQUES; HOMOLOGOUS RECOMBINATION; HYPOXANTHINE PHOSPHORIBOSYLTRANSFERASE; MALE; NUCLEAR TRANSFER TECHNIQUES; RABBITS;

EID: 84946751709     PISSN: None     EISSN: 20452322     Source Type: Journal    
DOI: 10.1038/srep16023     Document Type: Article

References (48)

1. Hooper, M., Hardy, K., Handyside, A., Hunter, S. & Monk, M. HPRT-deficient (Lesch-Nyhan) mouse embryos derived from germline colonization by cultured cells. Nature 326, 292-295 (1987)
2. Engle, S. J. et al. HPRT-APRT-deficient mice are not a model for lesch-nyhan syndrome. Hum Mol Genet 5, 1607-1610 (1996)
3. Schwiebert, E. M., Benos, D. J., Egan, M. E., Stutts, M. J. & Guggino, W. B. CFTR is a conductance regulator as well as a chloride channel. Physiol Rev 79, S145-166 (1999)
4. Evans, M. J. & Kaufman, M. H. Establishment in culture of pluripotential cells from mouse embryos. Nature 292, 154-156 (1981)
5. Martin, G. R. Isolation of a pluripotent cell line from early mouse embryos cultured in medium conditioned by teratocarcinoma stem cells. Proc Natl Acad Sci USA 78, 7634-7638 (1981)
6. Buehr, M. et al. Capture of authentic embryonic stem cells from rat blastocysts. Cell 135, 1287-1298 (2008)
7. Wilmut, I., Schnieke, A. E., McWhir, J., Kind, A. J. & Campbell, K. H. Viable offspring derived from fetal and adult mammalian cells. Nature 385, 810-813, doi: 10. 1038/385810a0 (1997)
8. Schnieke, A. E. et al. Human factor IX transgenic sheep produced by transfer of nuclei from transfected fetal fibroblasts. Science 278, 2130-2133 (1997)
9. Cibelli, J. B. et al. Cloned transgenic calves produced from nonquiescent fetal fibroblasts. Science 280, 1256-1258 (1998)
10. McCreath, K. J. et al. Production of gene-targeted sheep by nuclear transfer from cultured somatic cells. Nature 405, 1066-1069 (2000)
11. Lai, L. et al. Production of alpha-1, 3-galactosyltransferase knockout pigs by nuclear transfer cloning. Science 295, 1089-1092 (2002)
12. Rogers, C. S. et al. Production of CFTR-null and CFTR-DeltaF508 heterozygous pigs by adeno-associated virus-mediated gene targeting and somatic cell nuclear transfer. J Clin Invest 118, 1571-1577 (2008)
13. Sun, X. et al. Adeno-associated virus-targeted disruption of the CFTR gene in cloned ferrets. J Clin Invest 118, 1578-1583 (2008)
14. Hockemeyer, D. et al. Genetic engineering of human pluripotent cells using TALE nucleases. Nat Biotechnol 29, 731-734 (2012)
15. Tesson, L. et al. Knockout rats generated by embryo microinjection of TALENs. Nat Biotechnol 29, 695-696 (2011)
16. Song, J. et al. Generation of RAG 1- and 2-deficient rabbits by embryo microinjection of TALENs. Cell Res 23, 1059-1062 (2013)
17. Wu, Y. et al. Correction of a genetic disease in mouse via use of CRISPR-Cas9. Cell Stem Cell 13, 659-662 (2013)
18. Niu, Y. et al. Generation of Gene-Modified Cynomolgus Monkey via Cas9/RNA-Mediated Gene Targeting in One-Cell Embryos. Cell 156, 836-843 (2014)
19. Yang, D. et al. Effective gene targeting in rabbits using RNA-guided Cas9 nucleases. J Mol Cell Biol 6, 97-99 (2014)
20. Khan, I. F. et al. Engineering of human pluripotent stem cells by AAV-mediated gene targeting. Mol Ther 18, 1192-1199 (2010)
21. Fu, Y. et al. High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells. Nat Biotechnol 31, 822-826 (2013)
22. Chesne, P. et al. Cloned rabbits produced by nuclear transfer from adult somatic cells. Nat Biotechnol 20, 366-369 (2002)
23. Li, S., Chen, X., Fang, Z., Shi, J. & Sheng, H. Z. Rabbits generated from fibroblasts through nuclear transfer. Reproduction 131, 1085-1090, doi: 10. 1530/rep. 1. 01065 (2006)
24. Li, S. et al. Transgene expression of enhanced green fluorescent protein in cloned rabbits generated from in vitro-transfected adult fibroblasts. Transgenic Res 18, 227-235 (2009)
25. Zakhartchenko, V. et al. Cell-mediated transgenesis in rabbits: chimeric and nuclear transfer animals. Biol Reprod 84, 229-237 (2011)
26. Caskey, C. T. & Kruh, G. D. The HPRT locus. Cell 16, 1-9 (1979)
27. Melton, D. W., Konecki, D. S., Brennand, J. & Caskey, C. T. Structure, expression, and mutation of the hypoxanthine phosphoribosyltransferase gene. Proc Natl Acad Sci USA 81, 2147-2151 (1984)
28. Hirata, R., Chamberlain, J., Dong, R. & Russell, D. W. Targeted transgene insertion into human chromosomes by adenoassociated virus vectors. Nat Biotechnol 20, 735-738 (2002)
29. Russell, D. W. & Hirata, R. K. Human gene targeting by viral vectors. Nat Genet 18, 325-330 (1998)
30. Muzyczka, N. Use of adeno-associated virus as a general transduction vector for mammalian cells. Curr Top Microbiol Immunol 158, 97-129 (1992)
31. Stanford, W. L., Cohn, J. B. & Cordes, S. P. Gene-trap mutagenesis: past, present and beyond. Nat Rev Genet 2, 756-768 (2001)
32. Szymczak, A. L. & Vignali, D. A. Development of 2A peptide-based strategies in the design of multicistronic vectors. Expert Opin Biol Ther 5, 627-638 (2005)
33. Duda, K. et al. High-efficiency genome editing via 2A-coupled co-expression of fluorescent proteins and zinc finger nucleases or CRISPR/Cas9 nickase pairs. Nucleic Acids Res 42, e84 (2014)
34. Niwa, H. et al. An efficient gene-trap method using poly A trap vectors and characterization of gene-trap events. J Biochem 113, 343-349 (1993)
35. Flisikowska, T. et al. Efficient immunoglobulin gene disruption and targeted replacement in rabbit using zinc finger nucleases. PLoS One 6, e21045 (2011)
36. Yang, D. et al. Production of apolipoprotein C-III knockout rabbits using zinc finger nucleases. J Vis Exp. e50957 (2013)
37. Honda, A. et al. Single-step generation of rabbits carrying a targeted allele of the tyrosinase gene using CRISPR/Cas9. Exp Anim 64, 31-37 (2015)
38. Zambrowicz, B. P. et al. Wnk1 kinase deficiency lowers blood pressure in mice: a gene-trap screen to identify potential targets for therapeutic intervention. Proc Natl Acad Sci USA 100, 14109-14114 (2003)
39. Schnutgen, F. et al. Enhanced gene trapping in mouse embryonic stem cells. Nucleic Acids Res 36, e133 (2008)
40. Hanson, K. D. & Sedivy, J. M. Analysis of biological selections for high-efficiency gene targeting. Mol Cell Biol 15, 45-51 (1995)
41. Miller, D. G. AAV-mediated gene targeting. Methods Mol Biol 807, 301-315 (2011)
42. Topaloglu, O., Hurley, P. J., Yildirim, O., Civin, C. I. & Bunz, F. Improved methods for the generation of human gene knockout and knockin cell lines. Nucleic Acids Res 33, e158 (2005)
43. Niu, Y. et al. Generation of gene-modified cynomolgus monkey via Cas9/RNA-mediated gene targeting in one-cell embryos. Cell 156, 836-843 (2014)
44. Guibinga, G. H., Murray, F., Barron, N., Pandori, W. & Hrustanovic, G. Deficiency of the purine metabolic gene HPRT dysregulates microRNA-17 family cluster and guanine-based cellular functions: a role for EPAC in Lesch-Nyhan syndrome. Hum Mol Genet 22, 4502-4515 (2013)
45. Song, S. & Friedmann, T. Tissue-specific aberrations of gene expression in HPRT-deficient mice: functional complexity in a monogenic disease? Mol Ther 15, 1432-1443 (2007)
46. Lyon, M. F. Gene action in the X-chromosome of the mouse (Mus musculus L.). Nature 190, 372-373 (1961)
47. Okamoto, I. et al. Eutherian mammals use diverse strategies to initiate X-chromosome inactivation during development. Nature 472, 370-374 (2011)
48. Staessen, C., Janssenswillen, C., De Clerck, E. & Van Steirteghem, A. Controlled comparison of commercial media for human in-vitro fertilization: Menezo B2 medium versus Medi-Cult universal and BM1 medium. Hum Reprod 13, 2548-2554 (1998)