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Volumn 117, Issue 8, 2007, Pages 2233-2240

Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy

Author keywords

[No Author keywords available]

Indexed keywords

ADENOSINE DEAMINASE; BUSULFAN; CD34 ANTIGEN; RETROVIRUS VECTOR;

EID: 34547686397     PISSN: 00219738     EISSN: 15588238     Source Type: Journal    
DOI: 10.1172/JCI31666     Document Type: Article
Times cited : (228)

References (37)
  • 1
    • 0034724857 scopus 로고    scopus 로고
    • Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
    • Cavazzana-Calvo, M., et al. 2000. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science. 288:669-672.
    • (2000) Science , vol.288 , pp. 669-672
    • Cavazzana-Calvo, M.1
  • 2
    • 19944421384 scopus 로고    scopus 로고
    • Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector
    • Gaspar, H.B., et al. 2004. Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet. 364:2181-2187.
    • (2004) Lancet , vol.364 , pp. 2181-2187
    • Gaspar, H.B.1
  • 3
    • 0037189401 scopus 로고    scopus 로고
    • Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning
    • Aiuti, A., et al. 2002. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science. 296:2410-2413.
    • (2002) Science , vol.296 , pp. 2410-2413
    • Aiuti, A.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
    • 0036230462 scopus 로고    scopus 로고
    • Therapeutic applications for hematopoietic stem cell gene transfer
    • Bordignon, C., and Roncarolo, M.G. 2002. Therapeutic applications for hematopoietic stem cell gene transfer. Nat. Immunol. 3:318-321.
    • (2002) Nat. Immunol , vol.3 , pp. 318-321
    • Bordignon, C.1    Roncarolo, M.G.2
  • 6
    • 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
  • 7
    • 19344375031 scopus 로고    scopus 로고
    • Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences
    • Mitchell, R.S., et al. 2004. Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences. PLoS Biol. 2:e234.
    • (2004) PLoS Biol , vol.2
    • Mitchell, R.S.1
  • 8
    • 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
  • 9
    • 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
  • 10
    • 7044228128 scopus 로고    scopus 로고
    • Insertion of retroviral vectors in NOD/SCID repopulating human peripheral blood progenitor cells occurs preferentially in the vicinity of transcription start regions and in introns
    • Laufs, S., et al. 2004. Insertion of retroviral vectors in NOD/SCID repopulating human peripheral blood progenitor cells occurs preferentially in the vicinity of transcription start regions and in introns. Mol. Ther. 10:874-881.
    • (2004) Mol. Ther , vol.10 , pp. 874-881
    • Laufs, S.1
  • 11
    • 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
  • 12
    • 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
  • 13
    • 27944449787 scopus 로고    scopus 로고
    • Insertional mutagenesis identifies genes that promote the immortalization of primary bone marrow progenitor cells
    • Du, Y., Jenkins, N.A., and Copeland, N.G. 2005. Insertional mutagenesis identifies genes that promote the immortalization of primary bone marrow progenitor cells. Blood. 106:3932-3939.
    • (2005) Blood , vol.106 , pp. 3932-3939
    • Du, Y.1    Jenkins, N.A.2    Copeland, N.G.3
  • 14
    • 21044448037 scopus 로고    scopus 로고
    • Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking
    • Kustikova, O., et al. 2005. Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking. Science. 308:1171-1174.
    • (2005) Science , vol.308 , pp. 1171-1174
    • Kustikova, O.1
  • 15
    • 34249890562 scopus 로고    scopus 로고
    • Gene therapy for severe combined immunodeficiency: Are we there yet?
    • doi:10.1172/JCI30953
    • Cavazzana-Calvo, M., and Fischer, A. 2007. Gene therapy for severe combined immunodeficiency: are we there yet? J. Clin. Invest. 117:1456-1465. doi:10.1172/JCI30953.
    • (2007) J. Clin. Invest , vol.117 , pp. 1456-1465
    • Cavazzana-Calvo, M.1    Fischer, A.2
  • 16
    • 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
  • 17
    • 0347634456 scopus 로고    scopus 로고
    • Gene therapy insertional mutagenesis insights
    • Dave, U.P., Jenkins, N.A., and Copeland, N.G. 2004. Gene therapy insertional mutagenesis insights. Science. 303:333.
    • (2004) Science , vol.303 , pp. 333
    • Dave, U.P.1    Jenkins, N.A.2    Copeland, N.G.3
  • 19
    • 0031723552 scopus 로고    scopus 로고
    • Adenosine deaminase deficiency: Clinical expression, molecular basis, and therapy
    • Hershfield, M.S. 1998. Adenosine deaminase deficiency: clinical expression, molecular basis, and therapy. Semin. Hematol. 35:291-298.
    • (1998) Semin. Hematol , vol.35 , pp. 291-298
    • Hershfield, M.S.1
  • 20
    • 18444376150 scopus 로고    scopus 로고
    • Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement
    • Aiuti, A., et al. 2002. Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement. Nat. Med. 8:423-425.
    • (2002) Nat. Med , vol.8 , pp. 423-425
    • Aiuti, A.1
  • 21
    • 0036725051 scopus 로고    scopus 로고
    • New genes involved in cancer identified by retroviral tagging
    • Suzuki, T., et al. 2002. New genes involved in cancer identified by retroviral tagging. Nat. Genet. 32:166-174.
    • (2002) Nat. Genet , vol.32 , pp. 166-174
    • Suzuki, T.1
  • 22
    • 0034069495 scopus 로고    scopus 로고
    • Gene ontology: Tool for the unification of biology. The Gene Ontology Consortium
    • Ashburner, M., et al. 2000. Gene ontology: tool for the unification of biology. The Gene Ontology Consortium. Nat. Genet. 25:25-29.
    • (2000) Nat. Genet , vol.25 , pp. 25-29
    • Ashburner, M.1
  • 23
    • 0028789792 scopus 로고
    • Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients
    • Bordignon, C., et al. 1995. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients. Science. 270:470-475.
    • (1995) Science , vol.270 , pp. 470-475
    • Bordignon, C.1
  • 24
    • 0345144017 scopus 로고    scopus 로고
    • + cells from the cord blood of ADA-deficient SCID neonates
    • + cells from the cord blood of ADA-deficient SCID neonates. Nat. Med. 9:463-468.
    • (2003) Nat. Med , vol.9 , pp. 463-468
    • Schmidt, M.1
  • 25
    • 20144387186 scopus 로고    scopus 로고
    • + cells with lymphomyeloid differentiation potential and self-renewal capacity in the SCID-X1 gene therapy trial
    • + 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
  • 26
    • 31944440000 scopus 로고    scopus 로고
    • Retroviral vector integration deregulates gene expression but has no consequence on the biology and function of transplanted T cells
    • Recchia, A., et al. 2006. Retroviral vector integration deregulates gene expression but has no consequence on the biology and function of transplanted T cells. Proc. Natl. Acad. Sci. U. S. A. 103:1457-1462.
    • (2006) Proc. Natl. Acad. Sci. U. S. A , vol.103 , pp. 1457-1462
    • Recchia, A.1
  • 27
    • 33645451483 scopus 로고    scopus 로고
    • Mutagenesis and oncogenesis by chromosomal insertion of gene transfer vectors
    • Baum, C., Kustikova, O., Modlich, U., Li, Z., and Fehse, B. 2006. Mutagenesis and oncogenesis by chromosomal insertion of gene transfer vectors. Hum. Gene Ther. 17:253-263.
    • (2006) Hum. Gene Ther , vol.17 , pp. 253-263
    • Baum, C.1    Kustikova, O.2    Modlich, U.3    Li, Z.4    Fehse, B.5
  • 28
    • 25144490586 scopus 로고    scopus 로고
    • Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells
    • Calmels, B., et al. 2005. Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells. Blood. 106:2530-2533.
    • (2005) Blood , vol.106 , pp. 2530-2533
    • Calmels, B.1
  • 29
    • 34249906359 scopus 로고    scopus 로고
    • Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy
    • doi:10.1172/JCI31659
    • 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. doi:10.1172/JCI31659.
    • (2007) J. Clin. Invest , vol.117 , pp. 2225-2232
    • Deichmann, A.1
  • 30
    • 34547657665 scopus 로고    scopus 로고
    • Hot spots of retroviral integration in human CD34+ hematopoietic cells
    • In press
    • Cattoglio, C., et al. 2007. Hot spots of retroviral integration in human CD34+ hematopoietic cells. Blood. In press.
    • (2007) Blood
    • Cattoglio, C.1
  • 31
    • 33745225238 scopus 로고    scopus 로고
    • Fragile sites are preferential targets for integrations of MLV vectors in gene therapy
    • Bester, A.C., et al. 2006. Fragile sites are preferential targets for integrations of MLV vectors in gene therapy. Gene Ther. 13:1057-1059.
    • (2006) Gene Ther , vol.13 , pp. 1057-1059
    • Bester, A.C.1
  • 32
    • 0037033440 scopus 로고    scopus 로고
    • V(D)J-mediated translocations in lymphoid neoplasms: A functional assessment of genomic instability by cryptic sites
    • Marculescu, R., Le, T., Simon, P., Jaeger, U., and Nadel, B. 2002. V(D)J-mediated translocations in lymphoid neoplasms: a functional assessment of genomic instability by cryptic sites. J. Exp. Med. 195:85-98.
    • (2002) J. Exp. Med , vol.195 , pp. 85-98
    • Marculescu, R.1    Le, T.2    Simon, P.3    Jaeger, U.4    Nadel, B.5
  • 33
    • 1342289322 scopus 로고    scopus 로고
    • Activation of the T-cell oncogene LMO2 after gene therapy for X-linked severe combined immunodeficiency
    • McCormack, M.P., and Rabbitts, T.H. 2004. Activation of the T-cell oncogene LMO2 after gene therapy for X-linked severe combined immunodeficiency. N. Engl. J. Med. 350:913-922.
    • (2004) N. Engl. J. Med , vol.350 , pp. 913-922
    • McCormack, M.P.1    Rabbitts, T.H.2
  • 34
    • 33746801652 scopus 로고    scopus 로고
    • Unique risk factors for insertional mutagenesis in a mouse model of XSCID gene therapy
    • Shou, Y., Ma, Z., Lu, T., and Sorrentino, B.P. 2006. Unique risk factors for insertional mutagenesis in a mouse model of XSCID gene therapy. Proc. Natl. Acad. Sci. U. S. A. 103:11730-11735.
    • (2006) Proc. Natl. Acad. Sci. U. S. A , vol.103 , pp. 11730-11735
    • Shou, Y.1    Ma, Z.2    Lu, T.3    Sorrentino, B.P.4
  • 35
    • 33748924668 scopus 로고    scopus 로고
    • Gene therapy: Is IL2RG oncogenic in T-cell development?
    • discussion E6-E7
    • Pike-Overzet, K., et al. 2006. Gene therapy: is IL2RG oncogenic in T-cell development? Nature. 443:E5; discussion E6-E7.
    • (2006) Nature , vol.443
    • Pike-Overzet, K.1
  • 36
    • 33748939587 scopus 로고    scopus 로고
    • Gene therapy: X-SCID transgene leukaemogenicity
    • discussion E6-E7
    • Thrasher, A.J., et al. 2006. Gene therapy: X-SCID transgene leukaemogenicity. Nature. 443:E5-E6; discussion E6-E7.
    • (2006) Nature , vol.443
    • Thrasher, A.J.1
  • 37
    • 0002234927 scopus 로고    scopus 로고
    • Many multipotential gene-marked progenitor or stem cell clones contribute to hematopoiesis in nonhuman primates
    • Kim, H.J., et al. 2000. Many multipotential gene-marked progenitor or stem cell clones contribute to hematopoiesis in nonhuman primates. Blood. 96:1-8.
    • (2000) Blood , vol.96 , pp. 1-8
    • Kim, H.J.1


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.