Correction of a genetic defect in multipotent germline stem cells using a human artificial chromosome

Gene Therapy

Volumn 15, Issue 8, 2008, Pages 617-624

  Kazuki, Y ^a   Hoshiya, H ^a   Kai, Y ^a   Abe, S ^a   Takiguchi, M ^a   Osaki, M ^a   Kawazoe, S ^a   Katoh, M ^b   Kanatsu Shinohara, M ^c   Inoue, K ^d   Kajitani, N ^a   Yoshino, T ^a   Shirayoshi, Y ^a   Ogura, A ^d   Shinohara, T ^c   Barrett, J C ^e   Oshimura, M ^a  

^a TOTTORI UNIVERSITY   (Japan)

^b TOTTORI UNIVERSITY   (Japan)

^c KYOTO UNIVERSITY   (Japan)

^d INST OF PHYS AND CHEMICAL RESEARCH   (Japan)

^e NATIONAL CANCER INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

PROTEIN P53;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; ARTIFICIAL CHROMOSOME; CELL ASSAY; CELL DIFFERENTIATION; CELL SURVIVAL; CONTROLLED STUDY; GENE CONTROL; GENETIC DISORDER; GENOME ANALYSIS; GERM LINE GENE THERAPY; IN VITRO STUDY; IN VIVO STUDY; MOUSE; MULTIPOTENT STEM CELL; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PHENOTYPE; PRIORITY JOURNAL; RADIATION EXPOSURE; RADIOSENSITIVITY; ANIMAL; CANCER TRANSPLANTATION; CELL CULTURE; CHO CELL; CRICETULUS; CYTOLOGY; EMBRYONIC STEM CELL; FEMALE; FLUORESCENCE IN SITU HYBRIDIZATION; GENE EXPRESSION; GENE THERAPY; GENETIC TRANSFECTION; HAMSTER; HUMAN; MALE; METABOLISM; METHODOLOGY; MOLECULAR CLONING; TERATOMA; TRANSGENE; TRANSGENIC MOUSE; TUMOR SUPPRESSOR GENE;

ANIMALIA;

ANIMALS; CELL DIFFERENTIATION; CELLS, CULTURED; CHO CELLS; CHROMOSOMES, ARTIFICIAL, HUMAN; CLONING, MOLECULAR; CRICETINAE; CRICETULUS; EMBRYONIC STEM CELLS; FEMALE; GENE EXPRESSION; GENE THERAPY; GENES, P53; HUMANS; IN SITU HYBRIDIZATION, FLUORESCENCE; MALE; MICE; MICE, TRANSGENIC; MULTIPOTENT STEM CELLS; NEOPLASM TRANSPLANTATION; TERATOMA; TRANSFECTION; TRANSGENES;

EID: 43049168865     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/sj.gt.3303091     Document Type: Article

References (32)

1. Rideout III WM, Hochedlinger K, Kyba M, Daley GQ, Jaenisch R. Correction of a genetic defect by nuclear transplantation and combined cell and gene therapy. Cell 2002; 109: 17-27.
2. Kuroiwa Y, Kasinathan P, Matsushita H, Sathiyaselan J, Sullivan EJ, Kakitani M et al. Sequential targeting of the genes encoding immunoglobulin-mu and prion protein in cattle. Nat Genet 2004; 36: 775-780.
3. Cossu G, Sampaolesi M. New therapies for muscular dystrophy: Cautious optimism. Trends Mol Med 2004; 10: 516-520.
4. Odom GL, Gregorevic P, Chamberlain JS. Viral-mediated gene therapy for the muscular dystrophies: Successes, limitations and recent advances. Biochim Biophys Acta 2007; 1772: 243-262.
5. Pagani F, Buratti E, Stuani C, Bendix R, Dork T, Baralle FE. A new type of mutation causes a splicing defect in ATM. Nat Genet 2002; 30 426-429.
6. O'Connor TP, Crystal RG. Genetic medicines: Treatment strategies for hereditary disorders. Nat Rev Genet 2006; 7: 261-276.
7. Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003; 302 415-419.
8. Kuroiwa Y, Tomizuka K, Shinohara T, Kazuki Y, Yoshida H, Ohguma A et al. Manipulation ot human minichromosomes to carry greater than megabase-sized chromosome inserts. Nat Biotechnol 2000; 18: 1086-1090.
9. Katoh M, Ayabe F, Norikane S, Okada T, Masumoto H, Horike S et al. Construction of a novel human artificial chromosome vector for gene delivery. Biochem Biophys Res Commun 2004; 321: 280-290.
10. Basu J, Willard HF. Artificial and engineered chromosomes: non-integrating vectors for gene therapy. Trends Mol Med 2005; 11: 251-258.
11. Dennis C. Cloning7 mining the secrets of the egg. Nature 2006; 439: 652-655.
12. Hall VJ, Stojkovic P, Stojkovic M. Using therapeutic cloning to fight human disease: A conundrum or reality? Stem Cells 2006; 24: 1628-1637.
13. Kanatsu-Shinohara M, Inoue K, Lee J, Yoshimoto M, Ogonuki N, Miki H et al. Generation of pluripotent stem cells from neonatal mouse testis. Cell 2004; 119: 1001-1012.
14. Frengen E, Zhao B, Howe S, Weichenhan D, Osoegawa K, Gjernes E et al Modular bacterial artificial chromosome vectors for transfer of large inserts into mammalian cells. Genomics 2000; 68 118-126.
15. Horikawa I, Okamoto A, Yokota J, Oshimura M. Genetic heterogeneity of chromosome 11 associated with tumorigenicity in HeLa D98-OR cells. Cancer Genet Cytogenet 1995; 85: 97-100.
16. Ikeno M, Masumoto H, Okazaki T. Distribution of CENP-B boxes reflected in CREST centromere antigenic sites on long-range alpha-satellite DNA arrays of human chromosome 21. Hum Mol Genet 1994; 3: 1245-1257.
17. Kawasaki H, Mizuseki K, Nishikawa S, Kaneko S, Kuwana Y, Nakanishi S et al. Induction of midbrain dopaminergic neurons from ES cells by stromal cell-derived inducing activity. Neuron 2000; 28: 31-40.
18. Bourdon JC, Fernandes K, Murray-Zmijewski F, Liu G, Diot A, Xirodimas DP et al. p53 isoforms can regulate p53 transcriptional activity. Genes Dev 2005; 19: 2122-2137.
19. Tomizuka K, Yoshida H, Uejima H, Kugoh H, Sato K, Ohguma A et al. Functional expression and germline transmission of a human chromosome fragment in chimaeric mice. Nat Genet 1997; 16: 133-143.
20. Lin T, Chao C, Saito S, Mazur SJ, Murphy ME, Appella E et al. p53 induces differentiation of mouse embryonic stem cells by suppressing Nanog expression. Nat Cell Biol 2005; 7: 165-171.
21. Voet T, Schoenmakers E, Carpentier 5, Labaere C, Marynen P. Controlled transgene dosage and PAC-mediated transgenesis in mice using a chromosomal vector. Genomics 2003; 82: 596-605.
22. Mejia JE, Willmott A, Levy E, Earnshaw WC, Larin Z. Functional complementation of a genetic deficiency with human artificial chromosomes. Am J Hum Genet 2001; 69: 315-326.
23. Ayabe F, Katoh M, Inoue T, Kouprina N, Larionov V, Oshimura M. A novel expression system for genomic DNA loci using a human artificial chromosome vector with transformation-associated recombination cloning. J Hum Genet 2005; 50: 592-599.
24. Kouprina N, Larionov V. TAR cloning: Insights into gene function, long-range haplotypes and genome structure and evolution. Nat Rev Genet 2006; 7: 805-812.
25. Copeland NG, Jenkins NA, Court DL. Recombineering: A powerful new tool for mouse functional genomics. Nat Rev Genet 2001; 2: 769-779.
26. Wallace HA, Marques-Kranc F, Richardson M, Luna-Crespo F, Sharpe JA, Hughes J et al. Manipulating the mouse genome to engineer precise functional syntenic replacements with human sequence. Cell 2007; 128: 197-209.
27. Guan K, Nayernia K, Maier LS, Wagner S, Dressel R, Lee JH et al. Pluripotency of spermatogonial stem cells from adult mouse testis. Nature 2006; 440: 1199-1203.
28. Takahashi K, Yamanaka S. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell 2006; 126: 663-676.
29. Suda T, Katoh M, Hiratsuka M, Takiguchi M, Kazuki Y, Inoue T et al Heat-regulated production and secretion of insulin from a human artificial chromosome vector. Biochem Biophys Res Commun 2006; 340: 1053-1061.
30. Boley SE, Wong VA, French JE, Recio L. p53 heterozygosity alters the mRNA expression of p53 target genes in the bone marrow in response to inhaled benzene. Toxicol Sci 2002; 66: 209-215.
31. Otsuki A, Tahimic CG, Tomimatsu N, Katoh M, Chen DJ, Kurimasa A et al Construction of a novel expression system on a human artificial chromosome. Biochem Biophys Res Commun 2005; 329:1018-1025.
32. Trowell HE, Nagy A, Vissel B, Choo KH. Long-range analyses of the centromeric regions of human chromosomes 13, 14 and 21: Identification of a narrow domain containing two key centromeric DNA elements. Hum Mol Genet 1993; 2: 1639-1649.