Gene-delivery systems for iPS cell generation

Expert Opinion on Biological Therapy

Volumn 10, Issue 2, 2010, Pages 231-242

  Shao, Lijian ^a   Wu, Wen Shu ^a  

^a Maine Med Center Research Institute   (United States)

Author keywords

Embryonic stem cells; IPS cells; Pluripotent stem cells; Reprogramming

Indexed keywords

ADENOVIRUS VECTOR; LENTIVIRUS VECTOR; PLASMID VECTOR; RETROVIRUS VECTOR; TRANSPOSASE;

EPISOME; GENE DELIVERY SYSTEM; GENE EXPRESSION; GENE VECTOR; GENOME; HUMAN; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PLURIPOTENT STEM CELL; REVIEW; SOMATIC CELL; TRANSGENE; TRANSPOSON; VIRAL GENE DELIVERY SYSTEM;

ADENOVIRIDAE; ANIMALS; DNA TRANSPOSABLE ELEMENTS; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; PLASMIDS; PLURIPOTENT STEM CELLS; VIRUSES;

IPS;

EID: 76149112949     PISSN: 14712598     EISSN: None     Source Type: Journal    
DOI: 10.1517/14712590903455989     Document Type: Review

References (81)

1. Evans MJ, Kaufman MH. Establishment in culture of pluripotential cells from mouse embryos. Nature 1981;292(5819):154-156
2. Martin GR. Isolation of a pluripotent cell line from early mouse embryos cultured in medium conditioned by teratocarcinoma stem cells. Proc Natl Acad Sci USA 1981;78(12):7634-7638
3. Thomson JA, Itskovitz-Eldor J, Shapiro SS, et al. Embryonic stem cell lines derived from human blastocysts. Science 1998;282(5391):1145-1147
4. Blau CA. Current status of stem cell therapy and prospects for gene therapy for the disorders of globin synthesis. Baillieres Clin Haematol 1998;11(1):257-275
5. Moayeri M, Hawley TS, Hawley RG. Correction of murine hemophilia a by hematopoietic stem cell gene therapy. Mol Ther 2005;12(6):1034-1042
6. Burns CJ, Persaud SJ, Jones PM. Diabetes mellitus: a potential target for stem cell therapy. Curr Stem Cell Res Ther 2006;1(2):255-266
7. Yamaoka T. Regeneration therapy for diabetes mellitus. Expert Opin Biol Ther 2003;3(3):425-433
8. Kim JH, Auerbach JM, Rodriguez-Gomez JA, et al. Dopamine neurons derived from embryonic stem cells function in an animal model of parkinson's disease. Nature 2002;418(6893):50-56
9. Ho HY, Li M. Potential application of embryonic stem cells in parkinson's disease: drug screening and cell therapy. Regen Med 2006;1(2):175-182
10. Wernig M, Zhao JP, Pruszak J, et al. Neurons derived from reprogrammed fibroblasts functionally integrate into the fetal brain and improve symptoms of rats with parkinson's disease. Proc Natl Acad Sci USA 2008;105(15):5856-5861
11. Muramatsu S, Okuno T, Suzuki Y, et al. Multitracer assessment of dopamine function after transplantation of embryonic stem cell-derived neural stem cells in a primate model of parkinson's disease. Synapse 2009;63(7):541-548
12. Cicero SA, Johnson D, Reyntjens S, et al. Cells previously identified as retinal stem cells are pigmented ciliary epithelial cells. Proc Natl Acad Sci USA 2009;106(16):6685-6690
13. Matsuda R, Yoshikawa M, Kimura H, et al. Cotransplantation of mouse embryonic stem cells and bone marrow stromal cells following spinal cord injury suppresses tumor development. Cell Transplant 2009;18(1):39-54
14. Enzmann GU, Benton RL, Talbott JF, et al. Functional considerations of stem cell transplantation therapy for spinal cord repair. J Neurotrauma 2006;23(3-4):479-495
15. Hendricks WA, Pak ES, Owensby JP, et al. Predifferentiated embryonic stem cells prevent chronic pain behaviors and restore sensory function following spinal cord injury in mice. Mol Med 2006;12(1-3):34-46
16. McDonald JW, Becker D, Holekamp TF, et al. Repair of the injured spinal cord and the potential of embryonic stem cell transplantation. J Neurotrauma 2004;21(4):383-393
17. Kimura H, Yoshikawa M, Matsuda R, et al. Transplantation of embryonic stem cell-derived neural stem cells for spinal cord injury in adult mice. Neurol Res 2005;27(8):812-819
18. Markoulaki S, Meissner A, Jaenisch R. Somatic cell nuclear transfer and derivation of embryonic stem cells in the mouse. Methods 2008;45(2):101-114
19. Byrne JA. Generation of isogenic pluripotent stem cells. Hum Mol Genet 2008;17(R1):R37-41
20. Collas P, Taranger CK. Epigenetic reprogramming of nuclei using cell extracts. Stem Cell Rev 2006;2(4):309-317
21. Takahashi K, Yamanaka S. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell 2006;126(4):663-676
22. Okita K, Ichisaka T, Yamanaka S. Generation of germline-competent induced pluripotent stem cells. Nature 2007;448(7151):313-317
23. Wernig M, Meissner A, Foreman R, et al. In vitro reprogramming of fibroblasts into a pluripotent ES-cell-like state. Nature 2007;448(7151):318-324 (Pubitemid 47080337)
24. Takahashi K, Tanabe K, Ohnuki M, et al. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 2007;131(5):861-872
25. Yu J, Vodyanik MA, Smuga-Otto K, et al. Induced pluripotent stem cell lines derived from human somatic cells. Science 2007;318(5858):1917-1920
26. Shi Y, Desponts C, Do JT, et al. Induction of pluripotent stem cells from mouse embryonic fibroblasts by Oct4 and Klf4 with small-molecule compounds. Cell Stem Cell 2008;3(5):568-574
27. Shi Y, Do JT, Desponts C, et al. A combined chemical and genetic approach for the generation of induced pluripotent stem cells. Cell Stem Cell 2008;2(6):525-528
28. Huangfu D, Maehr R, Guo W, et al. Induction of pluripotent stem cells by defined factors is greatly improved by small-molecule compounds. Nat Biotechnol 2008;26(7):795-797
29. Silva J, Barrandon O, Nichols J, et al. Promotion of reprogramming to ground state pluripotency by signal inhibition. PLoS Biol 2008;6(10):e253. Published online 21 October 2008, doi:10.1371/journal.pbio.0060253
30. Zhou H, Wu S, Joo JY, et al. Generation of induced pluripotent stem cells using recombinant proteins. Cell Stem Cell 2009;4(5):381-384
31. Kim D, Kim CH, Moon JI, et al. Generation of human induced pluripotent stem cells by direct delivery of reprogramming proteins. Cell Stem Cell 2009;4(6):472-476
32. Aoi T, Yae K, Nakagawa M, et al. Generation of pluripotent stem cells from adult mouse liver and stomach cells. Science 2008;321(5889):699-702
33. Nakagawa M, Koyanagi M, Tanabe K, et al. Generation of induced pluripotent stem cells without Myc from mouse and human fibroblasts. Nat Biotechnol 2008;26(1):101-106
34. Park IH, Zhao R, West JA, et al. Reprogramming of human somatic cells to pluripotency with defined factors. Nature 2008;451(7175):141-146
35. Hotta A, Ellis J. Retroviral vector silencing during iPS cell induction: an epigenetic beacon that signals distinct pluripotent states. J Cell Biochem 2008;105(4):940-948
36. Aasen T, Raya A, Barrero MJ, et al. Efficient and rapid generation of induced pluripotent stem cells from human keratinocytes. Nat Biotechnol 2008;26(11):1276-1284
37. Kim JB, Zaehres H, Wu G, et al. Pluripotent stem cells induced from adult neural stem cells by reprogramming with two factors. Nature 2008;454(7204):646-650
38. Giorgetti A, Montserrat N, Aasen T, et al. Generation of induced pluripotent stem cells from human cord blood using OCT4 and SOX2. Cell Stem Cell 2009;5(4):353-357
39. Kim JB, Sebastiano V, Wu G, et al. Oct4-induced pluripotency in adult neural stem cells. Cell 2009;136(3):411-419
40. Hanna J, Wernig M, Markoulaki S, et al. Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin. Science 2007;318(5858):1920-1923
41. Xu D, Alipio Z, Fink LM, et al. Phenotypic correction of murine hemophilia A using an iPS cell-based therapy. Proc Natl Acad Sci USA 2009;106(3):808-813
42. Nelson TJ, Martinez-Fernandez A, Yamada S, et al. Repair of acute myocardial infarction with induced pluripotent stem cells induced by human stemness factors. Circulation 2009;120(5):408-416
43. Cattoglio C, Facchini G, Sartori D, et al. Hot spots of retroviral integration in human CD34+ hematopoietic cells. Blood 2007;110(6):1770-1778
44. Montini E, Cesana D, Schmidt M, et al. Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration. Nat Biotechnol 2006;24(6):687-696
45. Mali P, Ye Z, Hommond HH, et al. Improved efficiency and pace of generating induced pluripotent stem cells from human adult and fetal fibroblasts. Stem Cells 2008;26(8):1998-2005
46. Zhao Y, Yin X, Qin H, et al. Two supporting factors greatly improve the efficiency of human iPSC generation. Cell Stem Cell 2008;3(5):475-479
47. Brambrink T, Foreman R, Welstead GG, et al. Sequential expression of pluripotency markers during direct reprogramming of mouse somatic cells. Cell Stem Cell 2008;2(2):151-159
48. Stadtfeld M, Maherali N, Breault DT, et al. Defining molecular cornerstones during fibroblast to iPS cell reprogramming in mouse. Cell Stem Cell 2008;2(3):230-240
49. Maherali N, Ahfeldt T, Rigamonti A, et al. A high-efficiency system for the generation and study of human induced pluripotent stem cells. Cell Stem Cell 2008;3(3):340-345
50. Hockemeyer D, Soldner F, Cook EG, et al. A drug-inducible system for direct reprogramming of human somatic cells to pluripotency. Cell Stem Cell 2008;3(3):346-353
51. Shao L, Feng W, Sun Y, et al. Generation of iPS cells using defined factors linked via the self-cleaving 2a sequences in a single open reading frame. Cell Res 2009;19(3):296-306
52. Sommer CA, Stadtfeld M, Murphy GJ, et al. Induced pluripotent stem cell generation using a single lentiviral stem cell cassette. Stem Cells 2009;27(3):543-549
53. Chang CW, Lai YS, Pawlik KM, et al. Polycistronic lentiviral vector for "hit and run" Reprogramming of adult skin fibroblasts to induced pluripotent stem cells. Stem Cells 2009;27(5):1042-1049
54. Carey BW, Markoulaki S, Hanna J, et al. Reprogramming of murine and human somatic cells using a single polycistronic vector. Proc Natl Acad Sci USA 2009;106(1):157-162
55. Wernig M, Lengner CJ, Hanna J, et al. A drug-inducible transgenic system for direct reprogramming of multiple somatic cell types. Nat Biotechnol 2008;26(8):916-924
56. Okita K, Nakagawa M, Hyenjong H, et al. Generation of mouse induced pluripotent stem cells without viral vectors. Science 2008;322(5903):949-953
57. Gonzalez F, Barragan Monasterio M, Tiscornia G, et al. Generation of mouse-induced pluripotent stem cells by transient expression of a single nonviral polycistronic vector. Proc Natl Acad Sci USA 2009;106(22):8918-8922
58. Yates J, Warren N, Reisman D, et al. A cis-acting element from the epstein-barr viral genome that permits stable replication of recombinant plasmids in latently infected cells. Proc Natl Acad Sci USA 1984;81(12):3806- 3810
59. Yates JL, Warren N, Sugden B. Stable replication of plasmids derived from epstein-barr virus in various mammalian cells. Nature 1985;313(6005):812-815
60. Leight ER, Sugden B. Establishment of an oriP replicon is dependent upon an infrequent, epigenetic event. Mol Cell Biol 2001;21(13):4149-4161
61. Yates JL, Guan N. Epstein-Barr virus-derived plasmids replicate only once per cell cycle and are not amplified after entry into cells. J Virol 1991;65(1):483-488
62. Nanbo A, Sugden A, Sugden B. The coupling of synthesis and partitioning of EBV's plasmid replicon is revealed in live cells. EMBO J 2007;26(19):4252- 4262
63. Yu J, Hu K, Smuga-Otto K, et al. Human induced pluripotent stem cells free of vector and transgene sequences. Science 2009;324(5928):797-801
64. Stadtfeld M, Nagaya M, Utikal J, et al. Induced pluripotent stem cells generated without viral integration. Science 2008;322(5903):945-949
65. Zhou W, Freed CR. Adenoviral gene delivery can reprogram human fibroblasts to induced pluripotent stem cells. Stem Cells 2009;27(11):2667-2674
66. Soldner F, Hockemeyer D, Beard C, et al. Parkinson's disease patient-derived induced pluripotent stem cells free of viral reprogramming factors. Cell 2009;136(5):964-977
67. Mc Clintock B. The origin and behavior of mutable loci in maize. Proc Natl Acad Sci USA 1950;36(6):344-355
68. Cary LC, Goebel M, Corsaro BG, et al. Transposon mutagenesis of baculoviruses: analysis of trichoplusia ni transposon IFP2 insertions within the FP-locus of nuclear polyhedrosis viruses. Virology 1989;172(1):156-169
69. Fraser MJ, Ciszczon T, Elick T, et al. Precise excision of TTAA-specific lepidopteran transposons piggyBac (IFP2) and tagalong (TFP3) from the baculovirus genome in cell lines from two species of lepidoptera. Insect Mol Biol 1996;5(2):141-151
70. Woltjen K, Michael IP, Mohseni P, et al. piggyBac transposition reprograms fibroblasts to induced pluripotent stem cells. Nature 2009;458(7239):766-770
71. Kaji K, Norrby K, Paca A, et al. Virus-free induction of pluripotency and subsequent excision of reprogramming factors. Nature 2009;458(7239):771-775
72. Wang W, Lin C, Lu D, et al. Chromosomal transposition of piggyBac in mouse embryonic stem cells. Proc Natl Acad Sci USA 2008;105(27):9290-9295
73. Mermer B, Colb M, Krontiris TG. A family of short, interspersed repeats is associated with tandemly repetitive DNA in the human genome. Proc Natl Acad Sci USA 1987;84(10):3320-3324
74. Nelson TJ, Terzic A. Induced pluripotent stem cells: reprogrammed without a trace. Regen Med 2009;4(3):333-335
75. Leblois H, Roche C, Di Falco N, et al. Stable transduction of actively dividing cells via a novel adenoviral/episomal vector. Mol Ther 2000;1(4):314-322
76. Utikal J, Polo JM, Stadtfeld M, et al. Immortalization eliminates a roadblock during cellular reprogramming into iPS cells. Nature 2009;460(7259):1145-1148
77. Li H, Collado M, Villasante A, et al. The Ink4/Arf locus is a barrier for iPS cell reprogramming. Nature 2009;460(7259):1136-1139
78. Kawamura T, Suzuki J, Wang YV, et al. Linking the p53 tumour suppressor pathway to somatic cell reprogramming. Nature 2009;460(7259):1140-1144
79. Marion RM, Strati K, Li H, et al. A p53-mediated DNA damage response limits reprogramming to ensure iPS cell genomic integrity. Nature 2009;460(7259):1149-1153
80. Banito A, Rashid ST, Acosta JC, et al. Senescence impairs successful reprogramming to pluripotent stem cells. Genes Dev 2009;23(18):2134-2139
81. Hong H, Takahashi K, Ichisaka T, et al. Suppression of induced pluripotent stem cell generation by the p53-p21 pathway. Nature 2009;460(7259):1132-1135