Zinc finger nuclease-expressing baculoviral vectors mediate targeted genome integration of reprogramming factor genes to facilitate the generation of human induced pluripotent stem cells

Stem Cells Translational Medicine

Volumn 2, Issue 12, 2013, Pages 935-945

  Phang, Rui Zhe ^a   Tay, Felix Chang ^a   Goh, Sal Lee ^a   Lau, Cia Hin ^a   Zhu, Haibao ^a   Tan, Wee Kiat ^a   Liang, Qingle ^a   Chen, Can ^a   Du, Shouhui ^a   Li, Zhendong ^a   Tay, Johan Chin Kang ^a   Wu, Chunxiao ^b   Zeng, Jieming ^b   Fan, Weimin ^c   Toh, Han Chong ^d   Wang, Shu ^a,b  

^a NATIONAL UNIVERSITY OF SINGAPORE   (Singapore)

^b INSTITUTE OF BIOENGINEERING AND NANOTECHNOLOGY   (Singapore)

^c ZHEJIANG UNIVERSITY   (China)

^d DIVISION OF MEDICAL ONCOLOGY   (Singapore)

Author keywords

Cre loxP system; Gene delivery systems in vivo or in vitro; Gene expression; Induced pluripotent stem cells

Indexed keywords

BACULOVIRUS VECTOR; CD133 ANTIGEN; ELONGATION FACTOR 1ALPHA; HISTONE H2AX; KRUPPEL LIKE FACTOR 4; NESTIN; OCTAMER TRANSCRIPTION FACTOR 4; STAGE SPECIFIC EMBRYO ANTIGEN 4; TRANSCRIPTION FACTOR NANOG; TRANSCRIPTION FACTOR PAX6; TRANSCRIPTION FACTOR SOX1; TRANSCRIPTION FACTOR SOX2; ZINC FINGER NUCLEASE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CELL DIFFERENTIATION; CELL VIABILITY; CONTROLLED STUDY; CYTOTOXICITY; DNA SEQUENCE; FLOW CYTOMETRY; HISTOLOGY; HUMAN; HUMAN CELL; IMMUNOCYTOCHEMISTRY; IMMUNOFLUORESCENCE TEST; NONHUMAN; NUCLEAR REPROGRAMMING; ONCOGENE C MYC; PLURIPOTENT STEM CELL; PROTEIN EXPRESSION; REAL TIME POLYMERASE CHAIN REACTION; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; SOUTHERN BLOTTING; TERATOMA;

EID: 84888409717     PISSN: 21576564     EISSN: 21576580     Source Type: Journal    
DOI: 10.5966/sctm.2013-0043     Document Type: Article

References (33)

1. Robinton DA, Daley GQ. The promise of induced pluripotent stem cells in research and therapy. Nature 2012;481:295-305.
2. Sharkis SJ, Jones RJ, Civin C et al. Pluripotent stem cell-based cancer therapy: Promise and challenges. Sci Transl Med 2012;4:127ps9.
3. Schröder AR, Shinn P, Chen H et al. HIV-1 integration in the human genome favors active genes and local hotspots. Cell 2002;110:521-529.
4. Hacein-Bey-Abina S, Von Kalle C, Schmidt M et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003;302:415-419.
5. Collin J, Lako M et al. Robust, persistent transgene expression in human embryonic stem cells is achieved with AAVS1-targeted integration. STEM CELLS 2008;26:496-504.
6. Schambach A, Baum C. Clinical application of lentiviral vectors: Concepts and practice. Curr Gene Ther 2008;8:474-482.
7. Palpant NJ, Dudzinski D. Zinc finger nucleases: Looking toward translation. Gene Ther 2013;20:121-127.
8. Collin J, Lako M. Concise review: Putting a finger on stem cell biology: Zinc finger nuclease-driven targeted genetic editing in human pluripotent stem cells. STEM CELLS 2011;29: 1021-1033.
9. Cheng LT, Sun LT, Tada T. Genome editing in induced pluripotent stem cells. Genes Cells 2012;17:431-438.
10. Ramalingam S, London V, Kandavelou K et al. Generation and genetic engineering of human induced pluripotent stem cells using designed zinc finger nucleases. Stem Cells Dev 2013;22:595-610.
11. Kost TA, Condreay JP, Jarvis DL. Baculovirus as versatile vectors for protein expression in insect and mammalian cells. Nat Biotechnol 2005;23:567-575.
12. Chen CY, Lin CY, Chen GY et al. Baculovirus as a gene delivery vector: Recent understandings of molecular alterations in transduced cells and latest applications. Biotechnol Adv 2011;29:618-631.
13. DeKelver RC, Choi VM, Moehle EA et al. Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome. Genome Res 2010;20:1133-1142.
14. Hockemeyer D, Soldner F, Beard C et al. Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases. Nat Biotechnol 2009;27:851-857.
15. Miller JC et al. An improved zinc-finger nuclease architecture for highly specific genome editing. Nat Biotechnol 2007;25:778-785.
16. Ramachandra CJ, Shahbazi M, KwangTW et al. Efficient recombinase-mediated cassette exchange at the AAVS1 locus in human embryonic stem cells using baculoviral vectors. Nucleic Acids Res 2011;39:e107.
17. Rogakou EP, Pilch DR, Orr AH et al. DNA double-stranded breaks induce histone H2AX phosphorylation on serine 139. J Biol Chem 1998;273:5858-5868.
18. Rogakou EP, Boon C, Redon C et al. Megabase chromatin domains involved in DNA double-strand breaks in vivo. J Cell Biol 1999; 146:905-915.
19. Zeng J, Du J, Zhao Y et al. Baculoviral vector-mediated transient and stable transgene expression in human embryonic stem cells. STEM CELLS 2007;25:1055-1061.
20. Du J, Zeng J, Zhao Y et al. The combined use of viral transcriptional and post-transcriptional regulatory elements to improve baculovirus-mediated transient gene expression in human embryonic stem cells. J Biosci Bioeng 2010;109:1-8.
21. Sommer CA, Sommer AG, Longmire TA et al. Excision of reprogramming transgenes improves the differentiation potential of iPS cells generated with a single excisable vector. STEM CELLS 2010;28:64-74.
22. Ye L, Chang JC, Lin C et al. Generation of induced pluripotent stem cells using sitespecific integration with phage integrase. Proc Natl Acad Sci USA 2010;107:19467-19472.
23. Karow M, Chavez CL, Farruggio AP et al. Site-specific recombinase strategy to create induced pluripotent stem cells efficiently with plasmid DNA. STEM CELLS 2011;29:1696-1704.
24. Woltjen K, Michael IP, Mohseni P et al. piggyBac transposition reprograms fibroblasts to induced pluripotent stem cells. Nature 2009;458:766-770.
25. Grabundzija I, Wang J, Sebe A et al. Sleeping Beauty transposon-based system for cellular reprogramming and targeted gene insertion in induced pluripotent stem cells. Nucleic Acids Res 2013;41:1829-1847.
26. Takata Y, Kishine H, Sone T et al. Generation of iPS cells using a BacMam multigene expression system. Cell Struct Funct 2011;36: 209-222.
27. Gonza'lez F, Boue' S, Izpisúa Belmonte JC. Methods for making induced pluripotent stem cells: Reprogramming à la carte. Nat Rev Genet 2011;12:231-242.
28. Polo JM, Anderssen E, Walsh RM et al. A molecular roadmap of reprogramming somatic cells into iPS cells. Cell 2012;151:1617-1632.
29. Lei Y, Lee CL, Joo KI et al. Gene editing of human embryonic stem cells via an engineered baculoviral vector carrying zinc-finger nucleases. Mol Ther 2011;19:942-950.
30. Bak XY, Lam DH, Yang J et al. Human embryonic stem cell-derived mesenchymal stem cells as cellular delivery vehicles for prodrug gene therapy of glioblastoma. Hum Gene Ther 2011;22:1365-1377.
31. Zhao Y, Lam DH, Yang J et al. Targeted suicide gene therapy for glioma using human embryonic stem cell-derived neural stem cells genetically modified by baculoviral vectors. Gene Ther 2012;19:189-200.
32. Yang J, Lam DH, Goh SS et al. Tumor tropism of intravenously injected human-induced pluripotent stem cell-derived neural stem cells and their gene therapy application in a metastatic breast cancer model. STEM CELLS 2012;30: 1021-1029.
33. Du ZW, Hu BY, Ayala M et al. Cre recombination-mediated cassette exchange for building versatile transgenic human embryonic stem cells lines. STEM CELLS 2009;27: 1032-1041.