SEARCH

SEARCH BY CITATION

REFERENCES

  • 1
    Wu SM, Hochedlinger K. Harnessing the potential of induced pluripotent stem cells for regenerative medicine. Nat Cell Biol 2011; 13: 497505.
  • 2
    Sancho-Martinez I, Li M, Izpisua Belmonte JC. Disease correction the iPSC way: Advances in iPSC-based therapy. Clin Pharmacol Ther 2011; 89: 746749.
  • 3
    Ellis J. Silencing and variegation of gammaretrovirus and lentivirus vectors. Hum Gene Ther 2005; 16: 12411246.
  • 4
    Niwa O, Yokota Y, Ishida H et al. Independent mechanisms involved in suppression of the Moloney leukemia virus genome during differentiation of murine teratocarcinoma cells. Cell 1983; 32: 11051113.
  • 5
    Stewart CL, Stuhlmann H, Jahner D et al. De novo methylation, expression, and infectivity of retroviral genomes introduced into embryonal carcinoma cells. Proc Natl Acad Sci USA 1982; 79: 40984102.
  • 6
    Raya A, Rodriguez-Piza I, Guenechea G et al. Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells. Nature 2009; 460: 5359.
  • 7
    Wu G, Liu N, Rittelmeyer I et al. Generation of healthy mice from gene-corrected disease-specific induced pluripotent stem cells. PLoS Biol 2011; 9: e1001099.
  • 8
    Herbst F, Ball CR, Tuorto F et al. Extensive methylation of promoter sequences silences lentiviral transgene expression during stem cell differentiation in vivo. Mol Ther 2012; 20: 10141021.
  • 9
    Challita PM, Kohn DB. Lack of expression from a retroviral vector after transduction of murine hematopoietic stem cells is associated with methylation in vivo. Proc Natl Acad Sci USA 1994; 91: 25672571.
  • 10
    Klug CA, Cheshier S, Weissman IL. Inactivation of a GFP retrovirus occurs at multiple levels in long-term repopulating stem cells and their differentiated progeny. Blood 2000; 96: 894901.
  • 11
    Mukherjee S, Santilli G, Blundell MP et al. Generation of functional neutrophils from a mouse model of X-linked chronic granulomatous disorder using induced pluripotent stem cells. PLoS One 2011; 6: e17565.
  • 12
    Zou J, Sweeney CL, Chou BK et al. Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: Functional correction by zinc finger nuclease-mediated safe harbor targeting. Blood 2011; 117: 55615572.
  • 13
    Antoniou M, Harland L, Mustoe T et al. Transgenes encompassing dual-promoter CpG islands from the human TBP and HNRPA2B1 loci are resistant to heterochromatin-mediated silencing. Genomics 2003; 82: 269279.
  • 14
    Harland L, Crombie R, Anson S et al. Transcriptional regulation of the human TATA binding protein gene. Genomics 2002; 79: 479482.
  • 15
    Zhang F, Thornhill SI, Howe SJ et al. Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells. Blood 2007; 110: 14481457.
  • 16
    Williams S, Mustoe T, Mulcahy T et al. CpG-island fragments from the HNRPA2B1/CBX3 genomic locus reduce silencing and enhance transgene expression from the hCMV promoter/enhancer in mammalian cells. BMC Biotechnol 2005; 5: 17.
  • 17
    Zhang F, Frost AR, Blundell MP et al. A ubiquitous chromatin opening element (UCOE) confers resistance to DNA methylation-mediated silencing of lentiviral vectors. Mol Ther 2010; 18: 16401649.
  • 18
    Brendel C, Muller-Kuller U, Schultze-Strasser S et al. Physiological regulation of transgene expression by a lentiviral vector containing the A2UCOE linked to a myeloid promoter. Gene Ther 2012; 19: 10181029.
  • 19
    Moritz T, Williams DA. Marrow protection—Transduction of hematopoietic cells with drug resistance genes. Cytotherapy 2001; 3: 6784.
  • 20
    Flasshove M, Moritz T, Bardenheuer W et al. Hematoprotection by transfer of drug-resistance genes. Acta Haematol 2003; 110: 93106.
  • 21
    Bardenheuer W, Lehmberg K, Rattmann I et al. Resistance to cytarabine and gemcitabine and in vitro selection of transduced cells after retroviral expression of cytidine deaminase in human hematopoietic progenitor cells. Leukemia 2005; 19: 22812288.
  • 22
    Momparler RL, Eliopoulos N, Bovenzi V et al. Resistance to cytosine arabinoside by retrovirally mediated gene transfer of human cytidine deaminase into murine fibroblast and hematopoietic cells. Cancer Gene Ther 1996; 3: 331338.
  • 23
    Neff T, Blau CA. Forced expression of cytidine deaminase confers resistance to cytosine arabinoside and gemcitabine. Exp Hematol 1996; 24: 13401346.
  • 24
    Rattmann I, Kleff V, Sorg UR et al. Gene transfer of cytidine deaminase protects myelopoiesis from cytidine analogs in an in vivo murine transplant model. Blood 2006; 108: 29652971.
  • 25
    Brennig S, Rattmann I, Lachmann N et al. In vivo enrichment of cytidine deaminase gene-modified hematopoietic cells by prolonged cytosine-arabinoside application. Cytotherapy 2012; 14: 451460.
  • 26
    Lachmann N, Brennig S, Pfaff N et al. Efficient in vivo regulation of cytidine deaminase expression in the haematopoietic system using a doxycycline-inducible lentiviral vector system. Gene Ther 2012, doi: 10.1038/gt.2012.40 [Epub ahead of print, in press].
  • 27
    Baum C, Itoh K, Meyer J et al. The potent enhancer activity of the polycythemic strain of spleen focus-forming virus in hematopoietic cells is governed by a binding site for Sp1 in the upstream control region and by a unique enhancer core motif, creating an exclusive target for PEBP/CBF. J Virol 1997; 71: 63236331.
  • 28
    Milsom MD, Jerabek-Willemsen M, Harris CE et al. Reciprocal relationship between O6-methylguanine-DNA methyltransferase P140K expression level and chemoprotection of hematopoietic stem cells. Cancer Res 2008; 68: 61716180.
  • 29
    Schambach A, Bohne J, Baum C et al. Woodchuck hepatitis virus post-transcriptional regulatory element deleted from X protein and promoter sequences enhances retroviral vector titer and expression. Gene Ther 2006; 13: 641645.
  • 30
    Dull T, Zufferey R, Kelly M et al. A third-generation lentivirus vector with a conditional packaging system. J Virol 1998; 72: 84638471.
  • 31
    Pfaff N, Lachmann N, Kohlscheen S et al. Efficient hematopoietic redifferentiation of induced pluripotent stem cells derived from primitive murine bone marrow cells. Stem Cells Dev 2011; 21: 689701.
  • 32
    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: 89188922.
  • 33
    Lachmann N, Jagielska J, Heckl D et al. MicroRNA-150-regulated vectors allow lymphocyte-sparing transgene expression in hematopoietic gene therapy. Gene Ther 2012; 19: 915924.
  • 34
    Bock C, Reither S, Mikeska T et al. BiQ Analyzer: Visualization and quality control for DNA methylation data from bisulfite sequencing. Bioinformatics 2005; 21: 40674068.
  • 35
    Pfaffl MW. A new mathematical model for relative quantification in real-time RT-PCR. Nucleic Acids Res 2001; 29: e45.
  • 36
    Szabo PE, Hubner K, Scholer H et al. Allele-specific expression of imprinted genes in mouse migratory primordial germ cells. Mech Dev 2002; 115: 157160.
  • 37
    Wiles MV, Keller G. Multiple hematopoietic lineages develop from embryonic stem (ES) cells in culture. Development 1991; 111: 259267.
  • 38
    Reubinoff BE, Pera MF, Fong CY et al. Embryonic stem cell lines from human blastocysts: Somatic differentiation in vitro. Nat Biotechnol 2000; 18: 399404.
  • 39
    Lindahl Allen M, Antoniou M. Correlation of DNA methylation with histone modifications across the HNRPA2B1-CBX3 ubiquitously-acting chromatin open element (UCOE). Epigenetics 2007; 2: 227236.
  • 40
    Ott MG, Schmidt M, Schwarzwaelder K et al. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med 2006; 12: 401409.
  • 41
    Stein S, Ott MG, Schultze-Strasser S et al. Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nat Med 2010; 16: 198204.
  • 42
    Knight S, Zhang F, Mueller-Kuller U et al. Safer, silencing-resistant lentiviral vectors: Optimization of the ubiquitous chromatin-opening element through elimination of aberrant splicing. J Virol 2012; 86: 90889095.
  • 43
    Hockemeyer D, Soldner F, Beard C et al. Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases. Nat Biotechnol 2009; 27: 851857.
  • 44
    Mussolino C, Cathomen T. On target? Tracing zinc-finger-nuclease specificity. Nat Methods 2011; 8: 725726.
  • 45
    Cermak T, Doyle EL, Christian M et al. Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting. Nucleic Acids Res 2011; 39: e82.
  • 46
    Turan S, Bode J. Site-specific recombinases: From tag-and-target- to tag-and-exchange-based genomic modifications. FASEB J 2011; 25: 40884107.
  • 47
    Turan S, Galla M, Ernst E et al. Recombinase-mediated cassette exchange (RMCE): Traditional concepts and current challenges. J Mol Biol 2011; 407: 193221.
  • 48
    Burt RK, Verda L, Kim DA et al. Embryonic stem cells as an alternate marrow donor source: Engraftment without graft-versus-host disease. J Exp Med 2004; 199: 895904.
  • 49
    Choi KD, Yu J, Smuga-Otto K et al. Hematopoietic and endothelial differentiation of human induced pluripotent stem cells. Stem Cells 2009; 27: 559567.
  • 50
    Ma F, Ebihara Y, Umeda K et al. Generation of functional erythrocytes from human embryonic stem cell-derived definitive hematopoiesis. Proc Natl Acad Sci USA 2008; 105: 1308713092.
  • 51
    Vodyanik MA, Bork JA, Thomson JA et al. Human embryonic stem cell-derived CD34+ cells: Efficient production in the coculture with OP9 stromal cells and analysis of lymphohematopoietic potential. Blood 2005; 105: 617626.