Safeguarding clinical translation of pluripotent stem cells with suicide genes

Organogenesis

Volumn 9, Issue 1, 2013, Pages 34-39

  Li, Weiqiang ^a,b   Xiang, Andy Peng ^a,b  

^a SUN YAT SEN UNIVERSITY   (China)

^b SUN YAT SEN UNIVERSITY   (China)

Author keywords

Induced pluripotent stem cells; Regenerative medicine; Stem cell therapy; Suicide gene; Vector

Indexed keywords

ADENOVIRUS VECTOR; BACULOVIRUS VECTOR; CHLORMETHINE; FLUCYTOSINE; GANCICLOVIR; GLUTAMIC ACID; LENTIVIRUS VECTOR; OCTAMER TRANSCRIPTION FACTOR 4; PARVOVIRUS VECTOR; RETROVIRUS VECTOR; TRETAZICAR;

EPSTEIN BARR VIRUS; GENE VECTOR; HERPES SIMPLEX VIRUS; NONVIRAL GENE THERAPY; PLURIPOTENT STEM CELL; PROMOTER REGION; REGENERATIVE MEDICINE; REVIEW; STEM CELL GENE THERAPY; SUICIDE GENE; TRANSIENT EXPRESSION; CYTOLOGY; GENETICS; HUMAN; METABOLISM; STEM CELL TRANSPLANTATION; SUICIDE TRANSGENE; TRANSLATIONAL RESEARCH; VECTOR;

INDUCED PLURIPOTENT STEM CELLS; REGENERATIVE MEDICINE; STEM CELL THERAPY; SUICIDE GENE; VECTOR;

GENES, TRANSGENIC, SUICIDE; GENETIC VECTORS; HUMANS; PLURIPOTENT STEM CELLS; PROMOTER REGIONS, GENETIC; TRANSLATIONAL MEDICAL RESEARCH;

EID: 84876584474     PISSN: 15476278     EISSN: 15558592     Source Type: Journal    
DOI: 10.4161/org.24317     Document Type: Review

References (59)

1. Weissman IL. Stem cells: units of development, units of regeneration, and units in evolution. Cell 2000; 100:157-68; PMID:10647940. (Pubitemid 30046304)
2. Keller G. Embryonic stem cell differentiation: emergence of a new era in biology and medicine. Genes Dev 2005; 19:1129-55; PMID:15905405. (Pubitemid 40696181)
3. Hyun I. The bioethics of stem cell research and therapy. J Clin Invest 2010; 120:71-5; PMID:20051638.
4. Byrne JA, Pedersen DA, Clepper LL, Nelson M, Sanger WG, Gokhale S, et al. Producing primate embryonic stem cells by somatic cell nuclear transfer. Nature 2007; 450:497-502; PMID:18004281. (Pubitemid 350190255)
5. Cowan CA, Atienza J, Melton DA, Eggan K. Nuclear reprogramming of somatic cells after fusion with human embryonic stem cells. Science 2005; 309:1369-73; PMID:16123299. (Pubitemid 41209363)
6. Takahashi K, Yamanaka S. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell 2006; 126:663-76; PMID:16904174. (Pubitemid 44233629)
7. Takahashi K, Tanabe K, Ohnuki M, Narita M, Ichisaka T, Tomoda K, et al. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 2007; 131:861-72; PMID:18035408. (Pubitemid 350138099)
8. Yu J, Vodyanik MA, Smuga-Otto K, Antosiewicz-Bourget J, Frane JL, Tian S, et al. Induced pluripotent stem cell lines derived from human somatic cells. Science 2007; 318:1917-20; PMID:18029452.
9. Robinton DA, Daley GQ. The promise of induced pluripotent stem cells in research and therapy. Nature 2012; 481:295-305; PMID:22258608.
10. Narsinh K, Narsinh KH, Wu JC. Derivation of human induced pluripotent stem cells for cardiovascular disease modeling. Circ Res 2011; 108:1146-56; PMID:21527744.
11. Ben-David U, Benvenisty N. The tumorigenicity of human embryonic and induced pluripotent stem cells. Nat Rev Cancer 2011; 11:268-77; PMID:21390058.
12. Taapken SM, Nisler BS, Newton MA, Sampsell-Barron TL, Leonhard KA, McIntire EM, et al. Karotypic abnormalities in human induced pluripotent stem cells and embryonic stem cells. Nat Biotechnol 2011; 29:313-4; PMID:21478842.
13. Gore A, Li Z, Fung HL, Young JE, Agarwal S, Antosiewicz-Bourget J, et al. Somatic coding mutations in human induced pluripotent stem cells. Nature 2011; 471:63-7; PMID:21368825.
14. Tong M, Lv Z, Liu L, Zhu H, Zheng QY, Zhao XY, et al. Mice generated from tetraploid complementation competent iPS cells show similar developmental features as those from ES cells but are prone to tumori-genesis. Cell Res 2011; 21:1634-7; PMID:21876560.
15. Miura K, Okada Y, Aoi T, Okada A, Takahashi K, Okita K, et al. Variation in the safety of induced plu-ripotent stem cell lines. Nat Biotechnol 2009; 27:743-5; PMID:19590502.
16. Kiuru M, Boyer JL, O'Connor T P, Crystal RG. Genetic control of wayward pluripotent stem cells and their progeny after transplantation. Cell Stem Cell 2009; 4:289-300; PMID:19341619.
17. Yang NS, Sun WH. Gene gun and other non-viral approaches for cancer gene therapy. Nat Med 1995; 1:481-3; PMID:7585098.
18. Hewitt Z, Priddle H, Thomson AJ, Wojtacha D, McWhir J. Ablation of undifferentiated human embryonic stem cells: exploiting innate immunity against the Gal alpha1-3Galbeta1-4GlcNAc-R (alpha-Gal) epit-ope. Stem Cells 2007; 25:10-8; PMID:16960131. (Pubitemid 46052970)
19. Schepelmann S, Hallenbeck P, Ogilvie LM, Hedley D, Friedlos F, Martin J, et al. Systemic gene-directed enzyme prodrug therapy of hepatocellular carcinoma using a targeted adenovirus armed with car-boxypeptidase G2. Cancer Res 2005; 65:5003-8; PMID:15958540. (Pubitemid 40827305)
20. Hiraoka K, Kimura T, Logg CR, Kasahara N. Tumor-selective gene expression in a hepatic metastasis model after locoregional delivery of a replication-competent retrovirus vector. Clin Cancer Res 2006; 12:7108-16; PMID:17145835. (Pubitemid 44974511)
21. Schoensiegel F, Paschen A, Sieger S, Eskerski H, Mier W, Rothfels H, et al. MIA (melanoma inhibitory activity) promoter mediated tissue-specific suicide gene therapy of malignant melanoma. Cancer Gene Ther 2004; 11:408-18; PMID:15118759. (Pubitemid 38747372)
22. Yu D, Scott C, Jia WW, De Benedetti A, Williams BJ, Fazli L, et al. Targeting and killing of prostate cancer cells using lentiviral constructs containing a sequence recognized by translation factor eIF4E and a prostate-specific promoter. Cancer Gene Ther 2006; 13:32-43; PMID:16052226. (Pubitemid 41816347)
23. Paillard F. Epstein-Barr virus vectors for the treatment of Epstein-Barr virus-associated cancers. Hum Gene Ther 1998; 9:1119-20; PMID:9625250. (Pubitemid 28268997)
24. Detta A, Harland J, Hanif I, Brown SM, Cruickshank G. Proliferative activity and in vitro replication of HSV1716 in human metastatic brain tumours. J Gene Med 2003; 5:681-9; PMID:12898637. (Pubitemid 40309706)
25. Zhao Y, Lam DH, Yang J, Lin J, Tham CK, Ng WH, 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; PMID:21633393.
26. Norrman K, Fischer Y, Bonnamy B, Wolfhagen Sand F, Ravassard P, Semb H. Quantitative comparison of constitutive promoters in human ES cells. PLoS One 2010; 5:e12413; PMID:20865032.
27. Qin JY, Zhang L, Clift KL, Hulur I, Xiang A P, Ren BZ, et al. Systematic comparison of constitutive promoters and the doxycycline-inducible promoter. PLoS One 2010; 5:e10611; PMID:20485554.
28. Wu C, Lin J, Hong M, Choudhury Y, Balani P, Leung D, et al. Combinatorial control of suicide gene expression by tissue-specific promoter and microRNA regulation for cancer therapy. Mol Ther 2009; 17:2058-66; PMID:19809402.
29. Niess H, Bao Q, Conrad C, Zischek C, Notohamiprodjo M, Schwab F, et al. Selective targeting of genetically engineered mesenchymal stem cells to tumor stroma microenvironments using tissue-specific suicide gene expression suppresses growth of hepatocellular carcinoma. Ann Surg 2011; 254:767-74, discussion 774-5; PMID:22042469.
30. Goverdhana S, Puntel M, Xiong W, Zirger JM, Barcia C, Curtin J F, et al. Regulatable gene expression systems for gene therapy applications: progress and future challenges. Mol Ther 2005; 12:189-211; PMID:15946903. (Pubitemid 41025089)
31. Nakayama K, Pergolizzi RG, Crystal RG. Gene transfer-mediated pre-mRNA segmental trans-splicing as a strategy to deliver intracellular toxins for cancer therapy. Cancer Res 2005; 65:254-63; PMID:15665302. (Pubitemid 40070817)
32. Mazor R, Vassall AN, Eberle JA, Beers R, Weldon JE, Venzon DJ, et al. Identification and elimination of an immunodominant T-cell epitope in recombinant immunotoxins based on Pseudomonas exotoxin A. Proc Natl Acad Sci U S A 2012; 109:E3597-603; PMID:23213206.
33. Yamabe K, Shimizu S, Ito T, Yoshioka Y, Nomura M, Narita M, et al. Cancer gene therapy using a pro-apoptotic gene, caspase-3. Gene Ther 1999; 6:1952-9; PMID:10637446. (Pubitemid 30076865)
34. Carlotti F, Zaldumbide A, Martin P, Boulukos KE, Hoeben RC, Pognonec P. Development of an inducible suicide gene system based on human caspase 8. Cancer Gene Ther 2005; 12:627-39; PMID:15746943. (Pubitemid 40967223)
35. Springer CJ, Niculescu-Duvaz I. Prodrug-activating systems in suicide gene therapy. J Clin Invest 2000; 105: 1161-7 PMID:10791987. (Pubitemid 30266065)
36. Moolten FL. Tumor chemosensitivity conferred by inserted herpes thymidine kinase genes: paradigm for a prospective cancer control strategy. Cancer Res 1986; 46:5276-81; PMID:3019523. (Pubitemid 16008301)
37. Tiraby M, Cazaux C, Baron M, Drocourt D, Reynes J P, Tiraby G. Concomitant expression of E. coli cyto-sine deaminase and uracil phosphoribosyltransferase improves the cytotoxicity of 5-fluorocytosine. FEMS Microbiol Lett 1998; 167:41-9; PMID:9785450. (Pubitemid 28451607)
38. Huber BE, Richards CA, Krenitsky TA. Retroviral-mediated gene therapy for the treatment of hepatocel-lular carcinoma: an innovative approach for cancer therapy. Proc Natl Acad Sci U S A 1991; 88:8039-43; PMID:1654555. (Pubitemid 21915291)
39. Clark AJ, Iwobi M, Cui W, Crompton M, Harold G, Hobbs S, et al. Selective cell ablation in transgenic mice expression E. coli nitroreductase. Gene Ther 1997; 4:101-10; PMID:9081700. (Pubitemid 27096134)
40. Hedley D, Ogilvie L, Springer C. Carboxypeptidase-G2-based gene-directed enzyme-prodrug therapy: a new weapon in the GDEPT armoury. Nat Rev Cancer 2007; 7:870-9; PMID:17943135. (Pubitemid 350010854)
41. Jung J, Hackett NR, Pergolizzi RG, Pierre-Destine L, Krause A, Crystal RG. Ablation of tumor-derived stem cells transplanted to the central nervous system by genetic modification of embryonic stem cells with a suicide gene. Hum Gene Ther 2007; 18:1182-92; PMID:18021021. (Pubitemid 350261966)
42. Cao F, Lin S, Xie X, Ray P, Patel M, Zhang X, et al. In vivo visualization of embryonic stem cell survival, proliferation, and migration after cardiac delivery. Circulation 2006; 113:1005-14; PMID:16476845. (Pubitemid 43879476)
43. Schuldiner M, Itskovitz-Eldor J, Benvenisty N. Selective ablation of human embryonic stem cells expressing a "suicide" gene. Stem Cells 2003; 21:257-65; PMID:12743320. (Pubitemid 36623167)
44. Tang C, Lee AS, Volkmer J P, Sahoo D, Nag D, Mosley AR, et al. An antibody against SSEA-5 glycan on human pluripotent stem cells enables removal of teratoma-forming cells. Nat Biotechnol 2011; 29:829-34; PMID:21841799.
45. Wang Y, Yang D, Song L, Li T, Yang J, Zhang X, et al. Mifepristone-inducible caspase-1 expression in mouse embryonic stem cells eliminates tumor formation but spares differentiated cells in vitro and in vivo. Stem Cells 2012; 30:169-79; PMID:22131096.
46. Hara A, Aoki H, Taguchi A, Niwa M, Yamada Y, Kunisada T, et al. Neuron-like differentiation and selective ablation of undifferentiated embryonic stem cells containing suicide gene with Oct-4 promoter. Stem Cells Dev 2008; 17:619-27; PMID:18393636.
47. Naujok O, Kaldrack J, Taivankhuu T, Jörns A, Lenzen S. Selective removal of undifferentiated embryonic stem cells from differentiation cultures through HSV1 thymidine kinase and ganciclovir treatment. Stem Cell Rev 2010; 6:450-61; PMID:20411442.
48. Niwa H, Miyazaki J, Smith AG. Quantitative expression of Oct-3/4 defines differentiation, dedifferentiation or self-renewal of ES cells. Nat Genet 2000; 24:372-6; PMID:10742100. (Pubitemid 30187435)
49. Liedtke S, Stephan M, Kögler G. Oct4 expression revisited: potential pitfalls for data misinterpretation in stem cell research. Biol Chem 2008; 389:845-50; PMID:18627312.
50. Rong Z, Fu X, Wang M, Xu Y. A scalable approach to prevent teratoma formation of human embryonic stem cells. J Biol Chem 2012; 287:32338-45; PMID:22865887.
51. Zhong B, Watts KL, Gori JL, Wohlfahrt ME, Enssle J, Adair JE, et al. Safeguarding nonhuman primate iPS cells with suicide genes. Mol Ther 2011; 19:1667-75; PMID:21587213.
52. Cheng F, Ke Q, Chen F, Cai B, Gao Y, Ye C, et al. Protecting against wayward human induced pluripo-tent stem cells with a suicide gene. Biomaterials 2012; 33:3195-204; PMID:22269649.
53. Chen F, Cai B, Gao Y, Yuan X, Cheng F, Wang T, et al. Suicide gene-mediated ablation of tumor-initiating mouse pluripotent stem cells. Biomaterials 2013; 34:1701-11; PMID:23218839.
54. Salomon B, Maury S, Loubière L, Caruso M, Onclercq R, Klatzmann D. A truncated herpes simplex virus thy-midine kinase phosphorylates thymidine and nucleo-side analogs and does not cause sterility in transgenic mice. Mol Cell Biol 1995; 15:5322-8; PMID:7565681.
55. Chambers I, Colby D, Robertson M, Nichols J, Lee S, Tweedie S, et al. Functional expression cloning of Nanog, a pluripotency sustaining factor in embryonic stem cells. Cell 2003; 113:643-55; PMID:12787505. (Pubitemid 36694189)
56. Hockemeyer D, Soldner F, Beard C, Gao Q, Mitalipova M, DeKelver RC, et al. Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases. Nat Biotechnol 2009; 27:851-7; PMID:19680244.
57. Ding Q, Lee YK, Schaefer EA, Peters DT, Veres A, Kim K, et al. A TALEN Genome-Editing System for Generating Human Stem Cell-Based Disease Models. Cell Stem Cell 2013; 12:238-51; PMID:23246482.
58. Le Provost F, Lillico S, Passet B, Young R, Whitelaw B, Vilotte JL. Zinc finger nuclease technology heralds a new era in mammalian transgenesis. Trends Biotechnol 2010; 28:134-41; PMID:20015561.
59. Joung JK, Sander JD. TALENs: a widely applicable technology for targeted genome editing. Nat Rev Mol Cell Biol 2013; 14:49-55; PMID:23169466.