Generation of transgene-free human induced pluripotent stem cells with an excisable single polycistronic vector

Nature Protocols

Volumn 6, Issue 9, 2011, Pages 1251-1273

  Papapetrou, Eirini P ^a   Sadelain, Michel ^a  

^a MEMORIAL SLOAN KETTERING CANCER CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CRE RECOMBINASE; INTEGRASE; KRUPPEL LIKE FACTOR 4; LENTIVIRUS VECTOR; MYC PROTEIN; OCTAMER TRANSCRIPTION FACTOR 4; SINGLE POLYCISTRONIC LENTIVIRUS VECTOR; TRANSCRIPTION FACTOR SOX2; UNCLASSIFIED DRUG;

ARTICLE; CELL TYPE; CONTROLLED STUDY; GENE DOSAGE; GENE MAPPING; GENETIC SCREENING; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; HUMAN GENOME; INSERTIONAL MUTAGENESIS; LONG TERMINAL REPEAT; LOXP SITE; MESENCHYMAL STEM CELL; MUTAGENESIS; NONHUMAN; NUCLEAR REPROGRAMMING; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; PROTEIN ANALYSIS; PROTEIN EXPRESSION; TRANSGENE; TRANSIENT EXPRESSION;

ANIMALS; CELL CULTURE TECHNIQUES; CELL DIFFERENTIATION; CELLS, CULTURED; CULTURE MEDIA, CONDITIONED; GENETIC VECTORS; GENOME, HUMAN; HUMANS; INDUCED PLURIPOTENT STEM CELLS; KRUPPEL-LIKE TRANSCRIPTION FACTORS; LENTIVIRUS; MICE; OCTAMER TRANSCRIPTION FACTOR-3; PROTO-ONCOGENE PROTEINS C-MYC; SOXB1 TRANSCRIPTION FACTORS; TRANSGENES;

EID: 80052405826     PISSN: 17542189     EISSN: 17502799     Source Type: Journal    
DOI: 10.1038/nprot.2011.374     Document Type: Article

References (40)

1. Takahashi, K. & Yamanaka, S. Induction of pluripotent stem cells from mouse embryonic and adult fbroblast cultures by defned factors. Cell 126, 663-676 (2006). (Pubitemid 44233629)
2. Papapetrou, E.P. et al. Stoichiometric and temporal requirements of Oct4, Sox2, Klf4, and c-Myc expression for effcient human iPSC induction and differentiation. Proc. Natl. Acad. Sci. USA 106, 12759-12764 (2009).
3. Takahashi, K., Okita, K., Nakagawa, M. & Yamanaka, S. Induction of pluripotent stem cells from fbroblast cultures. Nat. Protoc. 2, 3081-3089 (2007).
4. Park, I.H., Lerou, P.H., Zhao, R., Huo, H. & Daley, G.Q. Generation of human-induced pluripotent stem cells. Nat. Protoc. 3, 1180-1186 (2008).
5. Raya, A. et al. A protocol describing the genetic correction of somatic human cells and subsequent generation of iPS cells. Nat. Protoc. 5, 647-660 (2010).
6. Aasen, T. & Belmonte, J.C. Isolation and cultivation of human keratinocytes from skin or plucked hair for the generation of induced pluripotent stem cells. Nat. Protoc. 5, 371-382 (2010).
7. Giorgetti, A. et al. Generation of induced pluripotent stem cells from human cord blood cells with only two factors: Oct4 and Sox2. Nat. Protoc. 5, 811-820 (2010).
8. Kim, J.B., Zaehres, H., Arauzo-Bravo, M.J. & Scholer, H.R. Generation of induced pluripotent stem cells from neural stem cells. Nat. Protoc. 4, 1464-1470 (2009).
9. Wernig, M. et al. In vitro reprogramming of fbroblasts into a pluripotent ES-cell-like state. Nature 448, 318-324 (2007). (Pubitemid 47080337)
10. Okita, K., Ichisaka, T. & Yamanaka, S. Generation of germline-competent induced pluripotent stem cells. Nature 448, 313-317 (2007). (Pubitemid 47080336)
11. Takahashi, K. et al. Induction of pluripotent stem cells from adult human fbroblasts by defned factors. Cell 131, 861-872 (2007). (Pubitemid 350138099)
12. Jaenisch, R. & Young, R. Stem cells, the molecular circuitry of pluripotency and nuclear reprogramming. Cell 132, 567-582 (2008). (Pubitemid 351253686)
13. Soldner, F. et al. Parkinson's disease patient-derived induced pluripotent stem cells free of viral reprogramming factors. Cell 136, 964-977 (2009).
14. Meissner, A., Wernig, M. & Jaenisch, R. Direct reprogramming of genetically unmodifed fbroblasts into pluripotent stem cells. Nat. Biotechnol. 25, 1177-1181 (2007). (Pubitemid 47538112)
15. Hu, B.Y. et al. Neural differentiation of human induced pluripotent stem cells follows developmental principles but with variable potency. Proc. Natl. Acad. Sci. USA 107, 4335-4340 (2010).
16. Boulting, G.L. et al. A functionally characterized test set of human induced pluripotent stem cells. Nat. Biotechnol. 29, 279-286 (2011).
17. Papapetrou, E.P. et al. Genomic safe harbors permit high beta-globin transgene expression in thalassemia induced pluripotent stem cells. Nat. Biotechnol. 29, 73-78 (2011).
18. Szymczak, A.L. et al. Correction of multi-gene defciency in vivo using a single 'self-cleaving' 2A peptide-based retroviral vector. Nat. Biotechnol. 22, 589-594 (2004). (Pubitemid 38585911)
19. Saenz, D.T. et al. Unintegrated lentivirus DNA persistence and accessibility to expression in nondividing cells: analysis with class I integrase mutants. J. Virol. 78, 2906-2920 (2004). (Pubitemid 38314348)
20. Stadtfeld, M., Nagaya, M., Utikal, J., Weir, G. & Hochedlinger, K. Induced pluripotent stem cells generated without viral integration. Science 322, 945-949 (2008).
21. Okita, K., Hong, H., Takahashi, K. & Yamanaka, S. Generation of mouse-induced pluripotent stem cells with plasmid vectors. Nat. Protoc. 5, 418-428 (2010).
22. Yu, J. et al. Human induced pluripotent stem cells free of vector and transgene sequences. Science 324, 797-801 (2009).
23. Narsinh, K.H. et al. Generation of adult human induced pluripotent stem cells using nonviral minicircle DNA vectors. Nat. Protoc. 6, 78-88 (2010).
24. Howden, S.E. et al. Genetic correction and analysis of induced pluripotent stem cells from a patient with gyrate atrophy. Proc. Natl. Acad. Sci. USA 108, 6537-6542 (2011).
25. Fusaki, N., Ban, H., Nishiyama, A., Saeki, K. & Hasegawa, M. Effcient induction of transgene-free human pluripotent stem cells using a vector based on Sendai virus, an RNA virus that does not integrate into the host genome. Proc. Jpn. Acad. Ser. B Phys. Biol. Sci. 85, 348-362 (2009).
26. Nishimura, K. et al. Development of defective and persistent sendai virus vector: a unique gene delivery/expression system ideal for cell reprogramming. J. Biol. Chem. 286, 4760-4771 (2011).
27. Warren, L. et al. Highly effcient reprogramming to pluripotency and directed differentiation of human cells with synthetic modifed mRNA. Cell Stem Cell 7, 618-630 (2010).
28. Zhou, H. et al. Generation of induced pluripotent stem cells using recombinant proteins. Cell Stem Cell 4, 381-384 (2009).
29. Kim, D. et al. Generation of human induced pluripotent stem cells by direct delivery of reprogramming proteins. Cell Stem Cell 4, 472-476 (2009).
30. Cho, H.J. et al. Induction of pluripotent stem cells from adult somatic cells by protein-based reprogramming without genetic manipulation. Blood 116, 386-395 (2010).
31. Kaji, K. et al. Virus-free induction of pluripotency and subsequent excision of reprogramming factors. Nature 458, 771-775 (2009).
32. Chang, C.W. et al. Polycistronic lentiviral vector for 'hit and run' reprogramming of adult skin fbroblasts to induced pluripotent stem cells. Stem Cells 27, 1042-1049 (2009).
33. Sommer, C.A. et al. Excision of reprogramming transgenes improves the differentiation potential of iPS cells generated with a single excisable vector. Stem Cells 28, 64-74 (2010).
34. Somers, A. et al. Generation of transgene-free lung disease-specifc human induced pluripotent stem cells using a single excisable lentiviral stem cell cassette. Stem Cells 28, 1728-1740 (2010).
35. Woltjen, K. et al. piggyBac transposition reprograms fbroblasts to induced pluripotent stem cells. Nature 458, 766-770 (2009).
36. Yusa, K., Rad, R., Takeda, J. & Bradley, A. Generation of transgene-free induced pluripotent mouse stem cells by the piggyBac transposon. Nat. Methods 6, 363-369 (2009).
37. Wang, W. et al. Chromosomal transposition of PiggyBac in mouse embryonic stem cells. Proc. Natl. Acad. Sci. USA 105, 9290-9295 (2008).
38. Huangfu, D. et al. Induction of pluripotent stem cells by defned factors is greatly improved by small-molecule compounds. Nat. Biotechnol. 26, 795-797 (2008). (Pubitemid 351961456)
39. Schmidt, M. et al. High-resolution insertion-site analysis by linear amplifcation-mediated PCR (LAM-PCR). Nat. Methods 4, 1051-1057 (2007). (Pubitemid 350201778)
40. Laurent, L.C. et al. Dynamic changes in the copy number of pluripotency and cell proliferation genes in human ESCs and iPSCs during reprogramming and time in culture. Cell Stem Cell 8, 106-118 (2011)