Artificial and engineered chromosomes: Non-integrating vectors for gene therapy

Trends in Molecular Medicine

Volumn 11, Issue 5, 2005, Pages 251-258

  Basu, Joydeep ^a   Willard, Huntington F ^a  

^a DUKE UNIVERSITY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BETA GLOBIN; BLOOD CLOTTING FACTOR 9; DNA VACCINE; DRUG CARRIER; ERYTHROPOIETIN; GUANOSINE TRIPHOSPHATE CYCLOHYDROLASE I; HERPESVIRUS VECTOR; HYPOXANTHINE PHOSPHORIBOSYLTRANSFERASE; IMMUNOGLOBULIN HEAVY CHAIN; IMMUNOGLOBULIN LAMBDA CHAIN; INTERLEUKIN 2; SATELLITE DNA; TRANSMEMBRANE CONDUCTANCE REGULATOR;

ARTIFICIAL CHROMOSOME; BACTERIAL ARTIFICIAL CHROMOSOME; CELL BASED GENE THERAPY; CELL COMPOSITION; CELL VIABILITY; CENTROMERE; CONSENSUS; DRUG EFFICACY; EPISOME; GENE AMPLIFICATION; GENE DELIVERY SYSTEM; GENE EXPRESSION; GENE TARGETING; GENE TRANSFER; GENETIC ENGINEERING; HUMAN; HUMAN CHROMOSOME; MALIGNANT TRANSFORMATION; MEDICAL ASSESSMENT; MEDICAL EXAMINATION; MINICHROMOSOME; NONHUMAN; REVIEW; TELOMERE; YEAST ARTIFICIAL CHROMOSOME;

CHROMOSOMES, ARTIFICIAL, HUMAN; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS;

EID: 18844363963     PISSN: 14714914     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.molmed.2005.03.006     Document Type: Review

References (74)

1. N. Somia, and I.M. Verma Gene therapy: trials and tribulations Nat. Rev. Genet. 1 2000 91 99
2. D.A. Williams, and C. Baum Gene therapy - new challenges ahead Science 302 2003 400 401
3. A. Recchia Site-specific integration of functional transgenes into the human genome by adeno/AAV hybrid vectors Mol. Ther. 10 2004 660 670
4. J.L. Portlock, and M.P. Calos Site-specific genomic strategies for gene therapy Curr. Opin. Mol. Ther. 5 2003 376 382
5. H. Caron The human transcriptome map: clustering of highly expressed genes in chromosomal domains Science 291 2001 1289 1292
6. R. Wade-Martins Long-term stability of large insert genomic DNA episomal shuttle vectors in human cells Nucleic Acids Res. 27 1999 1674 1682
7. H.J. Lipps Chromosome-based vectors for gene therapy Gene 304 2003 23 33
8. M. Conese Gene therapy progress and prospects: episomally maintained self-replicating systems Gene Ther. 11 2004 1735 1741
9. R. Wade-Martins Stable correction of a genetic deficiency in human cells by an episome carrying a 115 Kb genomic transgene Nat. Biotechnol. 18 2000 1311 1314
10. J. Black, and J.M. Vos Establishment of an oriP/EBNA1-based episomal vector transcribing human genomic β-globin in cultured murine fibroblasts Gene Ther. 9 2002 1447 1454
11. C.M. Chow LCR-mediated, long-term tissue-specific gene expression within replicating episomal plasmid and cosmid vectors Gene Ther. 9 2002 327 336
12. C.R. Sclimenti Epstein-Barr virus vectors provide prolonged robust factor IX expression in mice Biotechnol. Prog. 19 2003 144 151
13. R.E. White Infectious delivery of 120-kilobase genomic DNA by an Epstein-Barr virus amplicon vector Mol. Ther. 5 2002 427 435
14. R. Wade-Martins Infectious delivery of a 135-Kb LDLR genomic locus leads to regulated complementation of low-density lipoprotein receptor deficiency in human cells Mol. Ther. 7 2003 604 612
15. R. Inoue Infectious delivery of the 132 Kb CDKN2A/CDKN2B genomic DNA region results in correctly spliced gene expression and growth suppression in glioma cells Gene Ther. 11 2004 1195 1204
16. K.H. Choo Engineering human chromosomes for gene therapy studies Trends Mol. Med. 7 2001 235 237
17. W.R.A. Brown Artificial chromosomes: ideal vectors? Trends Biotechnol. 18 2000 218 223
18. D.W. Cleveland Centromeres and kinetochores: from epigenetics to mitotic checkpoint signaling Cell 112 2003 407 421
19. D.J. Amor Building the centromere: from foundation proteins to 3D organization Trends Cell Biol. 14 2004 359 368
20. H.F. Willard Centromeres: the missing link in the development of human artificial chromosomes Curr. Opin. Genet. Dev. 8 1998 219 225
21. M.G. Schueler Genomic and genetic definition of a functional human centromere Science 294 2001 109 115
22. M. Ikeno Construction of YAC-based mammalian artificial chromosomes Nat. Biotechnol. 16 1998 431 439
23. J.J. Harrington Formation of de novo centromeres and construction of first-generation human artificial microchromosomes Nat. Genet. 15 1997 345 355
24. R. Saffery Construction of neocentromere-based human minichromosomes by telomere-associated chromosomal truncation Proc. Natl. Acad. Sci. U. S. A. 98 2001 5705 5710
25. Q. Li Locus control regions Blood 100 2002 3077 3086
26. F. Recillas-Targa Prospects and implications of using chromatin insulators in gene therapy and transgenesis Bioessays 26 2004 796 807
27. J. Kereso De novo chromosome formations by large scale amplification of the centromeric region of mouse chromosomes Chromosome Res. 4 1996 226 239
28. G. Hadlaczky Satellite DNA-based artificial chromosomes for use in gene therapy Curr. Opin. Mol. Ther. 3 2001 125 132
29. D.O. Co Generation of transgenic mice and germline transmission of a mammalian artificial chromosome introduced into embryos by pronuclear microinjection Chromosome Res. 8 2000 183 191
30. M. Lindenbaum A mammalian artificial chromosome engineering system (ACE system) applicable to biopharmaceutical protein production, transgenesis and gene based cell therapy Nucleic Acids Res. 32 2004 e172
31. S. Stewart Retrofitting of a satellite repeat DNA-based murine artificial chromosome (ACes) to contain loxP recombination sites Gene Ther. 9 2002 719 723
32. S. Vanderbyl Transfer and stable transgene expression of a mammalian artificial chromosome into bone marrow-derived human mesenchymal stem cells Stem Cells 22 2004 324 333
33. H.F. Willard Chromosome manipulation: a systematic approach toward understanding human chromosome structure and function Proc. Natl. Acad. Sci. U. S. A. 93 1996 6847 6850
34. J.E. Itzhaki Targeted breakage of a human chromosome mediated by cloned, human telomeric DNA Nat. Genet. 2 1992 283 287
35. J.W. Yang Human mini-chromosomes with minimal centromeres Hum. Mol. Genet. 9 2000 1891 1902
36. M.H. Shen The accuracy of segregation of human mini-chromosomes varies in different vertebrate cell lines Chromosoma 109 2001 524 535
37. W. Mills Generation of an ∼2.4-Mb human X centromere-based minichromosome by targeted telomere-associated chromosome fragmentation in DT40 Hum. Mol. Genet. 8 1999 751 776
38. M. Otsuki Construction of a novel human artificial chromosome vector for gene delivery Biochem. Biophys. Res. Commun. 329 2005 1018 1025
39. M. Kakeda Human artificial chromosome (HAC) vector provides long-term therapeutic transgene expression in normal human primary fibroblasts Gene Ther 2005 10.1038/sj.gt.3302483 (http://www.nature.com/gt/)
40. D. Moralli Insertion of a loxP site in a size-reduced human accessory chromosome Cytogenet. Cell Genet. 94 2001 113 120
41. C. Auriche Functional human CFTR produced by a stable minichromosome EMBO Rep. 3 2002 862 868
42. Y. Kuroiwa Manipulation of human minichromosomes to carry greater than megabase-sized chromosome inserts Nat. Biotechnol. 18 2000 1086 1090
43. Y. Kuroiwa Cloned transchromosomic calves producing human immunoglobulin Nat. Biotechnol. 20 2002 889 894
44. J.M. Robl Artificial chromosome vectors and expression of complex proteins in transgenic animals Theriogenology 59 2003 107 113
45. C. Guiducci Use of a human mindchromosome as a cloning and expression vector for mammalian cells Hum. Mol. Genet. 8 1999 1417 1424
46. L.H. Wong Construction of neocentromere-based human minichromosomes for gene delivery and centromere studies Gene Ther. 9 2002 724 726
47. L.H. Wong Analysis of mitotic and expression properties of human neocentromere-based transchromosomes in mice J. Biol. Chem. 280 2004 3954 3962
48. A.W. Murray, and J.W. Szostak Construction of artificial chromosomes in yeast Nature 305 1983 189 193
49. T.A. Ebersole Mammalian artificial chromosome formation from circular alphoid input DNA does not require telomere repeats Hum. Mol. Genet. 9 2000 1623 1631
50. J.E. Mejia, and Z. Larin The assembly of large BACs by in vivo recombination Genomics 70 2000 165 170
51. J. Basu Rapid creation of BAC-based human artificial chromosome vectors by transposition with synthetic α-satellite arrays Nucleic Acids Res. 33 2005 587 596
52. J. Shampay DNA sequences of telomeres maintained in yeast Nature 310 1984 154 157
53. M.A. Barnett Telomere directed fragmentation of mammalian chromosomes Nucleic Acids Res. 21 1993 27 36
54. J.L. Hamlin Mammalian origins of replication Bioessays 14 1992 651 659
55. V. Todorovic Replication origins of mammalian chromosomes: the happy few Front. Biosci. 4 1999 D859 D868
56. M.I. Aladjem Genetic dissection of a mammalian replicator in the human β-globin locus Science 281 1998 1005 1009
57. K.A. Henning Human artificial chromosomes generated by modification of a yeast artificial chromosome containing both human α-satellite DNA and single copy sequences Proc. Natl. Acad. Sci. U. S. A. 96 1999 592 596
58. B.R. Grimes α-satellite DNA and vector composition influence rates of human artificial chromosome formation Mol. Ther. 5 2002 798 805
59. J.E. Mejia Efficiency of de novo centromere formation in human artificial chromosomes Genomics 79 2002 297 304
60. J. Ohzeki CENP-B box is required for de novo centromere chromatin assembly on human alphoid DNA J. Cell Biol. 159 2002 765 775
61. M.K. Rudd Human artificial chromosomes with α satellite-based de novo centromeres show increased frequency of nondisjunction and anaphase lag Mol. Cell. Biol. 23 2003 7689 7697
62. B.A. Sullivan, and H.F. Willard Stable dicentric X chromosomes with two functional centromeres Nat. Genet. 20 1998 227 228
63. Y. Muro Centromere protein B assembles human centromeric α-satellite DNA at the 17-bp sequence, CENP-B box J. Cell Biol. 116 1992 585 596
64. J.E. Mejia Functional complementation of a genetic deficiency with human artificial chromosomes Am. J. Hum. Genet. 69 2001 315 326
65. B.R. Grimes Stable gene expression from a mammalian artificial chromosome EMBO Rep. 2 2001 910 914
66. M. Ikeno Generation of human artificial chromosomes expressing naturally controlled guanosine triphosphate cyclohydrolase I gene Genes Cells 7 2002 1021 1032
67. C. Magin-Lachmann In vitro and in vivo delivery of intact BAC DNA - comparison of different methods J. Gene Med. 6 2004 195 209
68. G.D. Schmidt-Wolf, and I.G. Schmidt-Wolf Non-viral and hybrid vectors in human gene therapy: an update Trends Mol. Med. 9 2003 67 72
69. H.M. Byun Erythrocyte ghost-mediated gene delivery for prolonged and blood targeted expression Gene Ther. 11 2004 492 496
70. K. Tomizuka Double trans-chromosomic mice: maintenance of two individual human chromosome fragments containing Ig heavy and κ loci and expression of fully human antibodies Proc. Natl. Acad. Sci. U. S. A. 97 2000 722 727
71. A.M. Alazami Human artificial chromosomes containing chromosome 17 alphoid DNA maintain an active centromere in murine cells but are not stable Genomics 83 2004 844 851
72. K. Lundstrom Latest development in viral vectors for gene therapy Trends Biotechnol. 21 2003 117 122
73. S. Hacein-Bey-Abina LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1 Science 302 2003 415 419
74. L.E. Rosenberg, and A.N. Schechter Gene therapist, heal thyself Science 287 2000 1751