Stable maintenance of de novo assembled human artificial chromosomes in embryonic stem cells and their differentiated progeny in mice

Cell Cycle

Volumn 14, Issue 8, 2015, Pages 1268-1273

  Liskovykh, Mikhail ^a,b,c   Ponomartsev, Sergey ^a,b   Popova, Elena ^d   Bader, Michael ^d   Kouprina, Natalay ^c   Larionov, Vladimir ^c   Alenina, Natalia ^d   Tomilin, Alexey ^a,b  

^a INSTITUTE OF CYTOLOGY   (Russian Federation)

^b SKOLKOVO INSTITUTE OF SCIENCE AND TECHNOLOGY   (Russian Federation)

^c NATIONAL CANCER INSTITUTE   (United States)

^d MAX DELBRÜCK CENTER FOR MOLECULAR MEDICINE   (Germany)

Author keywords

embryonic stem cells; gene therapy; human artificial chromosomes

Indexed keywords

SYNTHETIC DNA;

ADULT; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CELL DIFFERENTIATION; CELL DIVISION; CONTROLLED STUDY; EMBRYO DEVELOPMENT; EMBRYONIC STEM CELL; EPISOME; FEMALE; FLUORESCENCE IN SITU HYBRIDIZATION; GENE EXPRESSION; GENETIC STABILITY; GENETIC TRANSCRIPTION; HUMAN; HUMAN ARTIFICIAL CHROMOSOME; HUMAN CELL; MALE; MOUSE; NONHUMAN; PLURIPOTENT STEM CELL; PROGENY; SOMATIC CELL; SUPERNUMERARY CHROMOSOME; ANIMAL; BLASTOCYST; C57BL MOUSE; CHO CELL LINE; CRICETULUS; CYTOLOGY; GENE THERAPY; GENE TRANSFER; GENETICS; HAMSTER; IMMUNOHISTOCHEMISTRY; METABOLISM; MOUSE EMBRYONIC STEM CELL; PATHOLOGY; TERATOMA;

ANIMALIA; MUS;

ANIMALS; BLASTOCYST; CELL DIFFERENTIATION; CHO CELLS; CHROMOSOMES, ARTIFICIAL, HUMAN; CRICETINAE; CRICETULUS; FEMALE; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; HUMANS; IMMUNOHISTOCHEMISTRY; IN SITU HYBRIDIZATION, FLUORESCENCE; MICE; MICE, INBRED C57BL; MOUSE EMBRYONIC STEM CELLS; TERATOMA;

EID: 84928038943     PISSN: 15384101     EISSN: 15514005     Source Type: Journal    
DOI: 10.1080/15384101.2015.1014151     Document Type: Article

References (27)

1. Hsu PD, Lander ES, Zhang F. Development and applications of CRISPR-Cas9 for genome engineering. Cell 2014; 157:1262-78; PMID:24906146; http://dx.doi.org/10.1016/j.cell.20l4.05.010
2. Kouprina N, Tomilin AN, Masumoto H, Earnshaw WC, Larionov V. Human artificial chromosome-based gene delivery vectors for biomedicine and biotechnology. Expert Opin Drug Deliv 2014; 11:517-35; PMID:24479793; http://dx.doi.org/10.1517/17425247.2014.882314
3. Kazuki Y, Hiratsuka M, Takiguchi M, Osaki M, Kaji-tani N, Hoshiya H, Hiramatsu K, Yoshino T, Kazuki K, Ishihara C, et al. Complete genetic correction of ips cells from Duchenne muscular dystrophy. Mol Ther 2010; 18:386-93; PMID:19997091; http://dx.doi.org/10.1038/mt.2009.274
4. Kazuki Y, Hoshiya H, Takiguchi M, Abe S, Iida Y, Osaki M, Katoh M, Hiratsuka M, Shirayoshi Y, Hira-matsu K, et al. Refined human artificial chromosome vectors for gene therapy and animal transgenesis. Gene Ther 2011; 18:384-93; PMID:21085194; http://dx. doi.org/10.1038/gt.2010.147
5. Yamaguchi S, Kazuki Y, Nakayama Y, Nanba E, Oshi-mura M, Ohbayashi T. A method for producing transgenic cells using a multi-integrase system on a human artificial chromosome vector. PLoS One 2011; 6: 17267; PMID:21390305; http://dx.doi.org/10.1371/journal.pone.0017267
6. Hoshiya H, Kazuki Y, Abe S, Takiguchi M, Kajitani N, Watanabe Y, Yoshino T, Shirayoshi Y, Higaki K, Messina G, et al. A highly stable and nonintegrated human artificial chromosome (HAC) containing the 2.4 Mb entire human dystrophin gene. Mol Ther 2009; 17:309-17; PMID:19034264; http://dx.doi.org/10.1038/mt.2008.253
7. Tedesco FS, Hoshiya H, D'Antona G, Gerli MF, Messina G, Antonini S, Tonlorenzi R, Benedetti S, Berghella L, Torrente Y, et al. Stem cell-mediated transfer ofa human artificial chromosome ameliorates muscular dystrophy. Sci Transl Med 2011; 3:96ra78; PMID:21849666; http://dx.doi.org/10.1126/scitranslmed.3002342
8. Tedesco FS, Gerli MF, Perani L, Benedetti S, Ungaro F, Cassano M, Antonini S, Tagliafico E, Artusi V, Longa E, et al. Transplantation of genetically corrected human iPSC-derived progenitors in mice with limb-girdle muscular dystrophy. Sci Transl Med 2012; 4:140ra89; PMID:22745439; http://dx.doi.org/10.1126/scitranslmed.3003541
9. Hiratsuka M, Uno N, Ueda K, Kurosaki H, Imaoka N, Kazuki K, Ueno E, Akakura Y, Katoh M, Osaki M, et al. Integration-free iPS cells engineered using human artificial chromosome vectors. PLoS One 2011; 6: 25961; PMID:21998730; http://dx.doi.org/10.1371/journal.pone.0025961
10. Ebersole T, Okamoto Y, Noskov VN, Kouprina N, Kim JH, Leem SH, Barrett JC, Masumoto H, Larionov V. Rapid generation of long synthetic tandem repeats and its application for analysis in human artificial chromosome formation. Nucleic Acids Res 2005; 33:130; PMID:16141190; http://dx.doi.org/10.1093/nar/gni129
11. Iida Y, Kim JH, Kazuki Y, Hoshiya H, Takiguchi M, Hayashi M, Erliandri I, Lee HS, Samoshkin A, Masu-moto H, et al. Human artificial chromosome with a conditional centromere for gene delivery and gene expression. DNA Res 2010; 17:293-301; PMID:20798231; http://dx.doi.org/10.1093/dnares/dsq020
12. Kim JH, Kononenko A, Erliandri I, Kim TA, Nakano M, Iida Y, Barrett JC, Oshimura M, Masumoto H,Earnshaw WC, et al. Human artificial chromosome (HAC) vector with a conditional centromere for correction ofgenetic deficiencies in human cells. Proceedings of the National Academy of Sciences of the United States ofAmerica 2011; 108:20048-53.
13. Nakano M, Cardinale S, Noskov VN, Gassmann R, Vagnarelli P, Kandels-Lewis S, Larionov V, Earnshaw WC, Masumoto H. Inactivation of a human kineto-chore by specific targeting of chromatin modifiers. Dev Cell 2008; 14:507-22; PMID:18410728; http://dx.doi.org/10.1016/j.devcel.2008.02.001
14. Kouprina N, Samoshkin A, Erliandri I, Nakano M, Lee HS, Fu H, Iida Y, Aladjem M, Oshimura H, Masu-moto H, et al. Organization ofsynthetic alphoid DNA array in human artificial chromosome (HAC) with a conditional centromere. ASC Synthetin Biology 2012; 1:590-601; PMID:23411994; http://dx.doi.org/10.1021/sb3000436
15. Lee NC, Kononenko AV, Lee HS, Tolkunova EN, Lis-kovykh MA, Masumoto H, Earnshaw WC, Tomilin AN, Larionov V, Kouprina N. Protecting a transgene expression from the HAC-based vector by different chromatin insulators. Cell Mol Life Sci 2013; 70:372-337; PMID:23677492; http://dx.doi.org/10.1007/s00018-013-1362-9
16. Kononenko AV, Bansal R, Lee NC, Grimes BR, Masumoto H, Earnshaw WC, Larionov V, Kouprina N. A portable BRCA1-HAC (human artificial chromosome) module for analysis of BRCA1 tumor suppressor function. Nucleic Acids Res 2014; 42; PMID: 25260588; http://dx.doi.org/10.1093/nar/gku870
17. Tomizuka K, Yoshida H, Uejima H, Kugoh H, Sato K, Ohguma A, Hayasaka M, Hanaoka K, Oshimura M, Ishida I. Functional expression and germline transmission of a human chromosome fragment in chimaeric mice. Nat Genet 1997; 16:133-43; PMID:9171824; http://dx.doi.org/10.1038/ng0697-133
18. Campos PB, Sartore RC, Abdalla SN, Rehen SK. Chromosomal spread preparation of human embryonic stem cells for karyotyping. J Vis Exp 2009; PMID:19734841
19. Liskovykh M, Chuykin I, Ranjan A, Safina D, Popova E, Tolkunova E, Mosienko V, Minina JM, Zhdanova NS, Mullins JJ, et al. Derivation, characterization, and stable transfection of induced pluripotent stem cells from Fischer344 rats. PLoS One 2011; 6:27345; PMID:22076153; http://dx.doi.org/10.1371/journal.pone.0027345
20. Kononenko AV, Lee NC, Liskovykh M, Masumoto H, Earnshaw WC, Larionov V, Kouprina N. Generation of a conditionally self-eliminating HAC gene delivery vector through incorporation ofa tTAVP64 expression cassette. Nucleic Acids Res 2015; PMID: 25712097; http://dx.doi.org/10.1093/nar/gkv124
21. Popova E, Rentzsch B, Bader M, Krivokharchenko A. Generation and characterization of a GFP transgenic rat line for embryological research. Transgenic Res 2008; 17:955-63; PMID:18523856; http://dx.doi.org/10.1007/s11248-008-9189-0
22. Ebersole T, Kim JH, Samoshkin A, Kouprina N, Pavli-cek A, White RJ, Larionov V. tRNA genes protect a reporter gene from epigenetic silencing in mouse cells. Cell Cycle 2011; 10:2779-91; PMID:21822054; http://dx.doi.org/10.4161/cc.10.16.17092
23. Evans MJ, Kaufman MH. Establishment in culture of pluripotential cells from mouse embryos. Nature 1981; 292:154-6; PMID:7242681; http://dx.doi.org/10.1038/292154a0
24. 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; http://dx.doi.org/10.1016/j.cell.2006.07.024
25. Huch M, Boj SF, Clevers H. Lgr5(+) liver stem cells, hepatic organoids and regenerative medicine. Regenerative medicine 2013; 8:385-7; PMID:23826690; http://dx.doi.org/10.2217/rme.13.39
26. Sato T, Clevers H. Growing self-organizing mini-guts from a single intestinal stem cell: mechanism and applications. Science 2013; 340:1190-4; PMID:23744940; http://dx.doi.org/10.1126/science.1234852
27. Barker N, Rookmaaker MB, Kujala P, Ng A, Leush-acke M, Snippert H, van de Wetering M, Tan S, Van Es JH, Huch M, et al. Lgr5(+ve) stem/progenitor cells contribute to nephron formation during kidney development. Cell Rep 2012; 2:540-52; PMID:22999937; http://dx.doi.org/10.1016/jxelrep.2012.08.018