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Volumn 119, Issue 4, 2009, Pages 964-975

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

Author keywords

[No Author keywords available]

Indexed keywords

B RAF KINASE; CYCLIN DEPENDENT KINASE INHIBITOR 2A; LENTIVIRUS VECTOR; RETROVIRUS VECTOR; CDKN2A PROTEIN, MOUSE; PRIMER DNA;

EID: 65249101260     PISSN: 00219738     EISSN: 15588238     Source Type: Journal    
DOI: 10.1172/JCI37630     Document Type: Article
Times cited : (487)

References (67)
  • 1
    • 7044253288 scopus 로고    scopus 로고
    • Stem cell clonality and genotoxicity in hematopoietic cells: Gene activation side effects should be avoidable
    • von Kalle, C., et al. 2004. Stem cell clonality and genotoxicity in hematopoietic cells: gene activation side effects should be avoidable. Semin. Hematol. 41:303-318.
    • (2004) Semin. Hematol , vol.41 , pp. 303-318
    • von Kalle, C.1
  • 2
    • 0037443457 scopus 로고    scopus 로고
    • Side effects of retroviral gene transfer into hematopoietic stem cells
    • Baum, C., et al. 2003. Side effects of retroviral gene transfer into hematopoietic stem cells. Blood. 101:2099-2114.
    • (2003) Blood , vol.101 , pp. 2099-2114
    • Baum, C.1
  • 3
    • 0037448352 scopus 로고    scopus 로고
    • A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency
    • Hacein-Bey-Abina, S., et al. 2003. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N. Engl. J. Med. 348:255-256.
    • (2003) N. Engl. J. Med , vol.348 , pp. 255-256
    • Hacein-Bey-Abina, S.1
  • 4
    • 33645734405 scopus 로고    scopus 로고
    • Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1
    • Ott, M.G., et al. 2006. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat. Med. 12:401-409.
    • (2006) Nat. Med , vol.12 , pp. 401-409
    • Ott, M.G.1
  • 5
    • 33947210723 scopus 로고    scopus 로고
    • Importance of murine study design for testing toxicity of retroviral vectors in support of phase I trials
    • Will, E., et al. 2007. Importance of murine study design for testing toxicity of retroviral vectors in support of phase I trials. Mol. Ther. 15:782-791.
    • (2007) Mol. Ther , vol.15 , pp. 782-791
    • Will, E.1
  • 6
    • 33646876218 scopus 로고    scopus 로고
    • Gene therapy advances but struggles to interpret safety data in small animal models
    • Williams, D.A. 2006. Gene therapy advances but struggles to interpret safety data in small animal models. Mol. Ther. 13:1027-1028.
    • (2006) Mol. Ther , vol.13 , pp. 1027-1028
    • Williams, D.A.1
  • 7
    • 33745108790 scopus 로고    scopus 로고
    • Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration
    • Montini, E., et al. 2006. Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration. Nat. Biotechnol. 24:687-696.
    • (2006) Nat. Biotechnol , vol.24 , pp. 687-696
    • Montini, E.1
  • 8
    • 0035487104 scopus 로고    scopus 로고
    • The INK4a/ARF network in tumour suppression
    • Sherr, C.J. 2001. The INK4a/ARF network in tumour suppression. Nat. Rev. Mol. Cell Biol. 2:731-737.
    • (2001) Nat. Rev. Mol. Cell Biol , vol.2 , pp. 731-737
    • Sherr, C.J.1
  • 9
    • 0842346437 scopus 로고    scopus 로고
    • Principles of tumor suppression
    • Sherr, C.J. 2004. Principles of tumor suppression. Cell. 116:235-246.
    • (2004) Cell , vol.116 , pp. 235-246
    • Sherr, C.J.1
  • 10
    • 51349090473 scopus 로고    scopus 로고
    • Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
    • Hacein-Bey-Abina, S., et al. 2008. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J. Clin. Invest. 118:3132-3142.
    • (2008) J. Clin. Invest , vol.118 , pp. 3132-3142
    • Hacein-Bey-Abina, S.1
  • 11
    • 51349158298 scopus 로고    scopus 로고
    • Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCIDX1 patients
    • Howe, S.J., et al. 2008. Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCIDX1 patients. J. Clin. Invest. 118:3143-3150.
    • (2008) J. Clin. Invest , vol.118 , pp. 3143-3150
    • Howe, S.J.1
  • 12
    • 0034040751 scopus 로고    scopus 로고
    • Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences
    • Follenzi, A., Ailles, L.E., Bakovic, S., Geuna, M., and Naldini, L. 2000. Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences. Nat. Genet. 25:217-222.
    • (2000) Nat. Genet , vol.25 , pp. 217-222
    • Follenzi, A.1    Ailles, L.E.2    Bakovic, S.3    Geuna, M.4    Naldini, L.5
  • 13
    • 7144261084 scopus 로고    scopus 로고
    • Antigen-specific cytolysis by neutrophils and NK cells expressing chimeric immune receptors bearing zeta or gamma signaling domains
    • Roberts, M.R., et al. 1998. Antigen-specific cytolysis by neutrophils and NK cells expressing chimeric immune receptors bearing zeta or gamma signaling domains. J. Immunol. 161:375-384.
    • (1998) J. Immunol , vol.161 , pp. 375-384
    • Roberts, M.R.1
  • 14
    • 20044396722 scopus 로고    scopus 로고
    • Distinct genomic integration of MLV and SIV vectors in primate hematopoietic stem and progenitor cells
    • Hematti, P., et al. 2004. Distinct genomic integration of MLV and SIV vectors in primate hematopoietic stem and progenitor cells. PLoS Biol. 2:e423.
    • (2004) PLoS Biol , vol.2
    • Hematti, P.1
  • 15
    • 33745684269 scopus 로고    scopus 로고
    • Retroviral DNA integration: Viral and cellular determinants of target-site selection
    • Lewinski, M.K., et al. 2006. Retroviral DNA integration: viral and cellular determinants of target-site selection. PLoS Pathog. 2:e60.
    • (2006) PLoS Pathog , vol.2
    • Lewinski, M.K.1
  • 16
    • 0037162715 scopus 로고    scopus 로고
    • HIV-1 integration in the human genome favors active genes and local hotspots
    • Schroder, A.R., et al. 2002. HIV-1 integration in the human genome favors active genes and local hotspots. Cell. 110:521-529.
    • (2002) Cell , vol.110 , pp. 521-529
    • Schroder, A.R.1
  • 17
    • 0037841763 scopus 로고    scopus 로고
    • Transcription start regions in the human genome are favored targets for MLV integration
    • Wu, X., Li, Y., Crise, B., and Burgess, S.M. 2003. Transcription start regions in the human genome are favored targets for MLV integration. Science. 300:1749-1751.
    • (2003) Science , vol.300 , pp. 1749-1751
    • Wu, X.1    Li, Y.2    Crise, B.3    Burgess, S.M.4
  • 18
    • 0031743608 scopus 로고    scopus 로고
    • Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery
    • Zufferey, R., et al. 1998. Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J. Virol. 72:9873-9880.
    • (1998) J. Virol , vol.72 , pp. 9873-9880
    • Zufferey, R.1
  • 19
    • 0021270238 scopus 로고
    • Determination of the leukaemogenicity of a murine retrovirus by sequences within the long terminal repeat
    • Lenz, J., et al. 1984. Determination of the leukaemogenicity of a murine retrovirus by sequences within the long terminal repeat. Nature. 308:467-470.
    • (1984) Nature , vol.308 , pp. 467-470
    • Lenz, J.1
  • 20
    • 0029892554 scopus 로고    scopus 로고
    • Retroviral insertional mutagenesis as a strategy to identify cancer genes
    • Jonkers, J., and Berns, A. 1996. Retroviral insertional mutagenesis as a strategy to identify cancer genes. Biochim. Biophys. Acta. 1287:29-57.
    • (1996) Biochim. Biophys. Acta , vol.1287 , pp. 29-57
    • Jonkers, J.1    Berns, A.2
  • 21
    • 33750624015 scopus 로고    scopus 로고
    • Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity
    • Modlich, U., et al. 2006. Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity. Blood. 108:2545-2553.
    • (2006) Blood , vol.108 , pp. 2545-2553
    • Modlich, U.1
  • 22
    • 49449084991 scopus 로고    scopus 로고
    • Leukemia induction after a single retroviral vector insertion in Evi1 or Prdm16
    • Modlich, U., et al. 2008. Leukemia induction after a single retroviral vector insertion in Evi1 or Prdm16. Leukemia. 22:1519-1528.
    • (2008) Leukemia , vol.22 , pp. 1519-1528
    • Modlich, U.1
  • 23
    • 28144462457 scopus 로고    scopus 로고
    • Genome-wide analysis of retroviral DNA integration
    • Bushman, F., et al. 2005. Genome-wide analysis of retroviral DNA integration. Nat. Rev. Microbiol. 3:848-858.
    • (2005) Nat. Rev. Microbiol , vol.3 , pp. 848-858
    • Bushman, F.1
  • 24
    • 15244346492 scopus 로고    scopus 로고
    • Promoter trapping reveals significant differences in integration site selection between MLV and HIV vectors in primary hematopoietic cells
    • De Palma, M., et al. 2005. Promoter trapping reveals significant differences in integration site selection between MLV and HIV vectors in primary hematopoietic cells. Blood. 105:2307-2315.
    • (2005) Blood , vol.105 , pp. 2307-2315
    • De Palma, M.1
  • 25
    • 34547657665 scopus 로고    scopus 로고
    • Hot spots of retroviral integration in human CD34+ hematopoietic cells
    • Cattoglio, C., et al. 2007. Hot spots of retroviral integration in human CD34+ hematopoietic cells. Blood. 110:1770-1778.
    • (2007) Blood , vol.110 , pp. 1770-1778
    • Cattoglio, C.1
  • 26
    • 34547686397 scopus 로고    scopus 로고
    • Multilineage hematopoietic reconstitution without clonal selection in ADASCID patients treated with stem cell gene therapy
    • Aiuti, A., et al. 2007. Multilineage hematopoietic reconstitution without clonal selection in ADASCID patients treated with stem cell gene therapy. J. Clin. Invest. 117:2233-2240.
    • (2007) J. Clin. Invest , vol.117 , pp. 2233-2240
    • Aiuti, A.1
  • 27
    • 34249906359 scopus 로고    scopus 로고
    • Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy
    • Deichmann, A., et al. 2007. Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy. J. Clin. Invest. 117:2225-2232.
    • (2007) J. Clin. Invest , vol.117 , pp. 2225-2232
    • Deichmann, A.1
  • 28
    • 34547651095 scopus 로고    scopus 로고
    • Gammaretrovirusmediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo
    • Schwarzwaelder, K., et al. 2007. Gammaretrovirusmediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo. J. Clin. Invest. 117:2241-2249.
    • (2007) J. Clin. Invest , vol.117 , pp. 2241-2249
    • Schwarzwaelder, K.1
  • 29
    • 34547691354 scopus 로고    scopus 로고
    • Retroviral integration and human gene therapy
    • Bushman, F.D. 2007. Retroviral integration and human gene therapy. J. Clin. Invest. 117:2083-2086.
    • (2007) J. Clin. Invest , vol.117 , pp. 2083-2086
    • Bushman, F.D.1
  • 30
    • 0030808926 scopus 로고    scopus 로고
    • 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
    • Baum, C., et al. 1997. 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. 71:6323-6331.
    • (1997) J. Virol , vol.71 , pp. 6323-6331
    • Baum, C.1
  • 31
    • 0034322479 scopus 로고    scopus 로고
    • Context dependence of different modules for posttranscriptional enhancement of gene expression from retroviral vectors
    • Schambach, A., et al. 2000. Context dependence of different modules for posttranscriptional enhancement of gene expression from retroviral vectors. Mol. Ther. 2:435-445.
    • (2000) Mol. Ther , vol.2 , pp. 435-445
    • Schambach, A.1
  • 32
    • 0036725322 scopus 로고    scopus 로고
    • Genome-wide retroviral insertional tagging of genes involved in cancer in Cdkn2a-deficient mice
    • Lund, A.H., et al. 2002. Genome-wide retroviral insertional tagging of genes involved in cancer in Cdkn2a-deficient mice. Nat. Genet. 32:160-165.
    • (2002) Nat. Genet , vol.32 , pp. 160-165
    • Lund, A.H.1
  • 33
    • 0029993450 scopus 로고    scopus 로고
    • Role of the INK4a locus in tumor suppression and cell mortality
    • Serrano, M., et al. 1996. Role of the INK4a locus in tumor suppression and cell mortality. Cell. 85:27-37.
    • (1996) Cell , vol.85 , pp. 27-37
    • Serrano, M.1
  • 34
    • 36749052217 scopus 로고    scopus 로고
    • High-resolution insertionsite analysis by linear amplification-mediated PCR (LAM-PCR)
    • Schmidt, M., et al. 2007. High-resolution insertionsite analysis by linear amplification-mediated PCR (LAM-PCR). Nat. Methods. 4:1051-1057.
    • (2007) Nat. Methods , vol.4 , pp. 1051-1057
    • Schmidt, M.1
  • 37
    • 34247628486 scopus 로고    scopus 로고
    • Mutation analysis of Son of Sevenless in juvenile myelomonocytic leukemia
    • Kratz, C.P., et al. 2007. Mutation analysis of Son of Sevenless in juvenile myelomonocytic leukemia. Leukemia. 21:1108-1109.
    • (2007) Leukemia , vol.21 , pp. 1108-1109
    • Kratz, C.P.1
  • 38
    • 0031035044 scopus 로고    scopus 로고
    • The localization of the HRX/ ALL1 protein to specific nuclear subdomains is altered by fusion with its eps15 translocation partner
    • Rogaia, D., et al. 1997. The localization of the HRX/ ALL1 protein to specific nuclear subdomains is altered by fusion with its eps15 translocation partner. Cancer Res. 57:799-802.
    • (1997) Cancer Res , vol.57 , pp. 799-802
    • Rogaia, D.1
  • 39
    • 22444444402 scopus 로고    scopus 로고
    • Cancer gene discovery in solid tumours using transposonbased somatic mutagenesis in the mouse
    • Collier, L.S., Carlson, C.M., Ravimohan, S., Dupuy, A.J., and Largaespada, D.A. 2005. Cancer gene discovery in solid tumours using transposonbased somatic mutagenesis in the mouse. Nature. 436:272-276.
    • (2005) Nature , vol.436 , pp. 272-276
    • Collier, L.S.1    Carlson, C.M.2    Ravimohan, S.3    Dupuy, A.J.4    Largaespada, D.A.5
  • 40
    • 34249901549 scopus 로고    scopus 로고
    • Sotos syndrome is associated with leukemia/lymphoma
    • Martinez-Glez, V., and Lapunzina, P. 2007. Sotos syndrome is associated with leukemia/lymphoma. Am J. Med. Genet. A. 143A:1244-1245.
    • (2007) Am J. Med. Genet. A , vol.143 A , pp. 1244-1245
    • Martinez-Glez, V.1    Lapunzina, P.2
  • 41
    • 30044442930 scopus 로고    scopus 로고
    • Redefining the common insertion site
    • Wu, X., Luke, B.T., and Burgess, S.M. 2006. Redefining the common insertion site. Virology. 344:292-295.
    • (2006) Virology , vol.344 , pp. 292-295
    • Wu, X.1    Luke, B.T.2    Burgess, S.M.3
  • 42
    • 41149133413 scopus 로고    scopus 로고
    • Physiological promoters reduce the genotoxic risk of integrating gene vectors
    • Zychlinski, D., et al. 2008. Physiological promoters reduce the genotoxic risk of integrating gene vectors. Mol. Ther. 16:718-725.
    • (2008) Mol. Ther , vol.16 , pp. 718-725
    • Zychlinski, D.1
  • 43
    • 33847408501 scopus 로고    scopus 로고
    • Retroviral vector insertion sites associated with dominant hematopoietic clones mark "stemness" pathways
    • Kustikova, O.S., et al. 2007. Retroviral vector insertion sites associated with dominant hematopoietic clones mark "stemness" pathways. Blood. 109:1897-1907.
    • (2007) Blood , vol.109 , pp. 1897-1907
    • Kustikova, O.S.1
  • 44
    • 0142084745 scopus 로고    scopus 로고
    • LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1
    • Hacein-Bey-Abina, S., et al. 2003. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science. 302:415-419.
    • (2003) Science , vol.302 , pp. 415-419
    • Hacein-Bey-Abina, S.1
  • 45
    • 22444431606 scopus 로고    scopus 로고
    • Mammalian mutagenesis using a highly mobile somatic Sleeping Beauty transposon system
    • Dupuy, A.J., Akagi, K., Largaespada, D.A., Copeland, N.G., and Jenkins, N.A. 2005. Mammalian mutagenesis using a highly mobile somatic Sleeping Beauty transposon system. Nature. 436:221-226.
    • (2005) Nature , vol.436 , pp. 221-226
    • Dupuy, A.J.1    Akagi, K.2    Largaespada, D.A.3    Copeland, N.G.4    Jenkins, N.A.5
  • 46
    • 21144437399 scopus 로고    scopus 로고
    • Leukemias following retroviral transfer of multidrug resistance 1 (MDR1) are driven by combinatorial insertional mutagenesis
    • Modlich, U., et al. 2005. Leukemias following retroviral transfer of multidrug resistance 1 (MDR1) are driven by combinatorial insertional mutagenesis. Blood. 105:4235-4246.
    • (2005) Blood , vol.105 , pp. 4235-4246
    • Modlich, U.1
  • 47
    • 34547844150 scopus 로고    scopus 로고
    • Co-occurrence analysis of insertional mutagenesis data reveals cooperating oncogenes
    • de Ridder, J., et al. 2007. Co-occurrence analysis of insertional mutagenesis data reveals cooperating oncogenes. Bioinformatics. 23:i133-i141.
    • (2007) Bioinformatics , vol.23
    • de Ridder, J.1
  • 48
    • 43149084575 scopus 로고    scopus 로고
    • Large-scale mutagenesis in p19(ARF)- and p53-deficient mice identifies cancer genes and their collaborative networks
    • Uren, A.G., et al. 2008. Large-scale mutagenesis in p19(ARF)- and p53-deficient mice identifies cancer genes and their collaborative networks. Cell. 133:727-741.
    • (2008) Cell , vol.133 , pp. 727-741
    • Uren, A.G.1
  • 49
    • 0021123235 scopus 로고
    • Tissuespecific transcription preference as a determinant of cell tropism and leukaemogenic potential of murine retroviruses
    • Celander, D., and Haseltine, W.A. 1984. Tissuespecific transcription preference as a determinant of cell tropism and leukaemogenic potential of murine retroviruses. Nature. 312:159-162.
    • (1984) Nature , vol.312 , pp. 159-162
    • Celander, D.1    Haseltine, W.A.2
  • 50
    • 58049220057 scopus 로고    scopus 로고
    • Combinatorial incorporation of enhancer blocking components of the chicken {beta}-globin 5'HS4 and human T-cell receptor {alpha}/{delta} BEAD-1 insulators in self-inactivating retroviral vectors reduces their genotoxic potential
    • Ramezani, A., Hawley, T.S., and Hawley, R.G. 2008. Combinatorial incorporation of enhancer blocking components of the chicken {beta}-globin 5'HS4 and human T-cell receptor {alpha}/{delta} BEAD-1 insulators in self-inactivating retroviral vectors reduces their genotoxic potential. Stem Cells. 26:3257-3266.
    • (2008) Stem Cells , vol.26 , pp. 3257-3266
    • Ramezani, A.1    Hawley, T.S.2    Hawley, R.G.3
  • 51
    • 58149386433 scopus 로고    scopus 로고
    • Insertional gene activation by lentiviral and gammaretroviral vectors
    • Bokhoven, M., et al. 2009. Insertional gene activation by lentiviral and gammaretroviral vectors. J. Virol. 83:283-294.
    • (2009) J. Virol , vol.83 , pp. 283-294
    • Bokhoven, M.1
  • 52
    • 34249298695 scopus 로고    scopus 로고
    • Improving transcriptional termination of self-inactivating gamma-retroviral and lentiviral vectors
    • Schambach, A., Galla, M., Maetzig, T., Loew, R., and Baum, C. 2007. Improving transcriptional termination of self-inactivating gamma-retroviral and lentiviral vectors. Mol. Ther. 15:1167-1173.
    • (2007) Mol. Ther , vol.15 , pp. 1167-1173
    • Schambach, A.1    Galla, M.2    Maetzig, T.3    Loew, R.4    Baum, C.5
  • 53
    • 38049016006 scopus 로고    scopus 로고
    • Successful treatment of canine leukocyte adhesion deficiency by foamy virus vectors
    • Bauer, T.R., Jr., et al. 2008. Successful treatment of canine leukocyte adhesion deficiency by foamy virus vectors. Nat. Med. 14:93-97.
    • (2008) Nat. Med , vol.14 , pp. 93-97
    • Bauer Jr., T.R.1
  • 54
    • 50549096864 scopus 로고    scopus 로고
    • Reduced genotoxicity of avian sarcoma leukosis virus vectors in rhesus long-term repopulating cells compared to standard murine retrovirus vectors
    • Hu, J., et al. 2008. Reduced genotoxicity of avian sarcoma leukosis virus vectors in rhesus long-term repopulating cells compared to standard murine retrovirus vectors. Mol. Ther. 16:1617-1623.
    • (2008) Mol. Ther , vol.16 , pp. 1617-1623
    • Hu, J.1
  • 55
    • 1342311458 scopus 로고    scopus 로고
    • Sleeping beauty transposition: Biology and applications for molecular therapy
    • Izsvak, Z., and Ivics, Z. 2004. Sleeping beauty transposition: biology and applications for molecular therapy. Mol. Ther. 9:147-156.
    • (2004) Mol. Ther , vol.9 , pp. 147-156
    • Izsvak, Z.1    Ivics, Z.2
  • 56
    • 39549110741 scopus 로고    scopus 로고
    • An experimental system for the evaluation of retroviral vector design to diminish the risk for proto-oncogene activation
    • Ryu, B.Y., et al. 2008. An experimental system for the evaluation of retroviral vector design to diminish the risk for proto-oncogene activation. Blood. 111:1866-1875.
    • (2008) Blood , vol.111 , pp. 1866-1875
    • Ryu, B.Y.1
  • 57
    • 35948946526 scopus 로고    scopus 로고
    • Gene editing in human stem cells using zinc finger nucleases and integrasedefective lentiviral vector delivery
    • Lombardo, A., et al. 2007. Gene editing in human stem cells using zinc finger nucleases and integrasedefective lentiviral vector delivery. Nat. Biotechnol. 25:1298-1306.
    • (2007) Nat. Biotechnol , vol.25 , pp. 1298-1306
    • Lombardo, A.1
  • 58
    • 0037510038 scopus 로고    scopus 로고
    • Enhancing gene targeting with designed zinc finger nucleases
    • Bibikova, M., Beumer, K., Trautman, J.K., and Carroll, D. 2003. Enhancing gene targeting with designed zinc finger nucleases. Science. 300:764.
    • (2003) Science , vol.300 , pp. 764
    • Bibikova, M.1    Beumer, K.2    Trautman, J.K.3    Carroll, D.4
  • 60
    • 0029996147 scopus 로고    scopus 로고
    • In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector
    • Naldini, L., et al. 1996. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science. 272:263-267.
    • (1996) Science , vol.272 , pp. 263-267
    • Naldini, L.1
  • 61
    • 0032143165 scopus 로고    scopus 로고
    • Generating green fluorescent mice by germline transmission of green fluorescent ES cells
    • Hadjantonakis, A.K., Gertsenstein, M., Ikawa, M., Okabe, M., and Nagy, A. 1998. Generating green fluorescent mice by germline transmission of green fluorescent ES cells. Mech. Dev. 76:79-90.
    • (1998) Mech. Dev , vol.76 , pp. 79-90
    • Hadjantonakis, A.K.1    Gertsenstein, M.2    Ikawa, M.3    Okabe, M.4    Nagy, A.5
  • 62
    • 0034931366 scopus 로고    scopus 로고
    • A model for the detection of clonality in marked hematopoietic stem cells
    • discussion 155-146
    • Schmidt, M., et al. 2001. A model for the detection of clonality in marked hematopoietic stem cells. Ann. N. Y. Acad. Sci. 938:146-155; discussion 155-146.
    • (2001) Ann. N. Y. Acad. Sci , vol.938 , pp. 146-155
    • Schmidt, M.1
  • 63
    • 20144387186 scopus 로고    scopus 로고
    • Clonal evidence for the transduction of CD34+ cells with lymphomyeloid differentiation potential and self-renewal capacity in the SCID-X1 gene therapy trial
    • Schmidt, M., et al. 2005. Clonal evidence for the transduction of CD34+ cells with lymphomyeloid differentiation potential and self-renewal capacity in the SCID-X1 gene therapy trial. Blood. 105:2699-2706.
    • (2005) Blood , vol.105 , pp. 2699-2706
    • Schmidt, M.1
  • 64
    • 0034915569 scopus 로고    scopus 로고
    • Detection and direct genomic sequencing of multiple rare unknown flanking DNA in highly complex samples
    • Schmidt, M., et al. 2001. Detection and direct genomic sequencing of multiple rare unknown flanking DNA in highly complex samples. Hum. Gene Ther. 12:743-749.
    • (2001) Hum. Gene Ther , vol.12 , pp. 743-749
    • Schmidt, M.1
  • 65
    • 17344392308 scopus 로고    scopus 로고
    • A new mathematical model for relative quantification in real-time RT-PCR
    • Pfaffl, M.W. 2001. A new mathematical model for relative quantification in real-time RT-PCR. Nucleic Acids Res. 29:e45.
    • (2001) Nucleic Acids Res , vol.29
    • Pfaffl, M.W.1
  • 66
    • 27644482283 scopus 로고    scopus 로고
    • Comprehensive algorithm for quantitative real-time polymerase chain reaction
    • Zhao, S., and Fernald, R.D. 2005. Comprehensive algorithm for quantitative real-time polymerase chain reaction. J. Comput. Biol. 12:1047-1064.
    • (2005) J. Comput. Biol , vol.12 , pp. 1047-1064
    • Zhao, S.1    Fernald, R.D.2
  • 67
    • 45449087921 scopus 로고    scopus 로고
    • qBase relative quantification framework and software for management and automated analysis of real-time quantitative PCR data
    • Hellemans, J., Mortier, G., De Paepe, A., Speleman, F., and Vandesompele, J. 2007. qBase relative quantification framework and software for management and automated analysis of real-time quantitative PCR data. Genome Biol. 8:R19.
    • (2007) Genome Biol , vol.8
    • Hellemans, J.1    Mortier, G.2    De Paepe, A.3    Speleman, F.4    Vandesompele, J.5


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