메뉴 건너뛰기




Volumn 9, Issue 10, 2007, Pages 833-842

Gene therapy clinical trials worldwide to 2007 - An update

Author keywords

[No Author keywords available]

Indexed keywords

ANTIPARKINSON AGENT; CERE 120; GENDICINE; GENE THERAPY AGENT; GLUTAMATE DECARBOXYLASE; NEURTURIN; SMALL INTERFERING RNA; UNCLASSIFIED DRUG;

EID: 35148817461     PISSN: 1099498X     EISSN: 15212254     Source Type: Journal    
DOI: 10.1002/jgm.1100     Document Type: Review
Times cited : (490)

References (43)
  • 1
    • 4444370799 scopus 로고    scopus 로고
    • Gene therapy clinical trials worldwide 1989-2004 - an overview
    • Edelstein ML, Abedi MR, Wixon J, Edelstein RM. Gene therapy clinical trials worldwide 1989-2004 - an overview. J Gene Med 2004; 6: 597-602.
    • (2004) J Gene Med , vol.6 , pp. 597-602
    • Edelstein, M.L.1    Abedi, M.R.2    Wixon, J.3    Edelstein, R.M.4
  • 2
    • 0024988334 scopus 로고
    • Gene transfer into humans - immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction
    • Rosenberg SA, Aebersold P, Cornetta. K, et al. Gene transfer into humans - immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction. N Engl J Med 1990; 323: 570-578.
    • (1990) N Engl J Med , vol.323 , pp. 570-578
    • Rosenberg, S.A.1    Aebersold, P.2    Cornetta, K.3
  • 3
    • 0142089747 scopus 로고    scopus 로고
    • Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer
    • Raper SE, Chirmule N, Lee FS, et al. Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genet Metab 2003; 80: 148-158.
    • (2003) Mol Genet Metab , vol.80 , pp. 148-158
    • Raper, S.E.1    Chirmule, N.2    Lee, F.S.3
  • 4
    • 13944278472 scopus 로고    scopus 로고
    • As Gelsinger case ends, gene therapy suffers another blow
    • Couzin J, Kaiser J. As Gelsinger case ends, gene therapy suffers another blow. Science 2005; 307: 1028.
    • (2005) Science , vol.307 , pp. 1028
    • Couzin, J.1    Kaiser, J.2
  • 5
    • 0034724857 scopus 로고    scopus 로고
    • Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
    • Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 2000; 288: 669-672.
    • (2000) Science , vol.288 , pp. 669-672
    • Cavazzana-Calvo, M.1    Hacein-Bey, S.2    de Saint Basile, G.3
  • 6
    • 0037448352 scopus 로고    scopus 로고
    • A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency
    • Hacein-Bey-Abina S, von Kalle C, Schmidt M, et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2003; 348: 255-256.
    • (2003) N Engl J Med , vol.348 , pp. 255-256
    • Hacein-Bey-Abina, S.1    von Kalle, C.2    Schmidt, M.3
  • 7
    • 0142084745 scopus 로고    scopus 로고
    • LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1
    • Hacein-Bey-Abina S, Von Kalle C, Schmidt M, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003; 302: 415-419.
    • (2003) Science , vol.302 , pp. 415-419
    • Hacein-Bey-Abina, S.1    Von Kalle, C.2    Schmidt, M.3
  • 8
    • 19944421384 scopus 로고    scopus 로고
    • Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector
    • Gaspar HB, Parsley KL, Howe S, et al. Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet 2004; 364: 2181-2187.
    • (2004) Lancet , vol.364 , pp. 2181-2187
    • Gaspar, H.B.1    Parsley, K.L.2    Howe, S.3
  • 9
    • 20844459483 scopus 로고    scopus 로고
    • Treatment of an infant with X-linked severe combined immunodeficiency (SCID-X1) by gene therapy in Australia
    • Ginn SL, Curtin JA, Kramer B, et al. Treatment of an infant with X-linked severe combined immunodeficiency (SCID-X1) by gene therapy in Australia. Med J Aust 2005; 182: 458-463.
    • (2005) Med J Aust , vol.182 , pp. 458-463
    • Ginn, S.L.1    Curtin, J.A.2    Kramer, B.3
  • 10
    • 33748413936 scopus 로고    scopus 로고
    • Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning
    • Gaspar HB, Bjorkegren E, Parsley K, et al. Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning. Mol Ther 2006; 14: 505-513.
    • (2006) Mol Ther , vol.14 , pp. 505-513
    • Gaspar, H.B.1    Bjorkegren, E.2    Parsley, K.3
  • 11
    • 14044262371 scopus 로고    scopus 로고
    • Policy statement on the social, ethical and public awareness issues in gene therapy
    • for the European Society of Gene Therapy
    • Gansbacher B for the European Society of Gene Therapy. Policy statement on the social, ethical and public awareness issues in gene therapy. J Gene Med 2002; 4: 687-691.
    • (2002) J Gene Med , vol.4 , pp. 687-691
    • Gansbacher, B.1
  • 12
    • 0043033652 scopus 로고    scopus 로고
    • Report of a second serious adverse event in a clinical trial of gene therapy for X-linked severe combined immune deficiency (X-SCID). Position of the European Society of Gene Therapy (ESGT)
    • for the European Society of Gene Therapy
    • Gansbacher B for the European Society of Gene Therapy. Report of a second serious adverse event in a clinical trial of gene therapy for X-linked severe combined immune deficiency (X-SCID). Position of the European Society of Gene Therapy (ESGT). J Gene Med 2003; 5: 261-262.
    • (2003) J Gene Med , vol.5 , pp. 261-262
    • Gansbacher, B.1
  • 13
    • 35148870407 scopus 로고    scopus 로고
    • Report from the ASGT Ad Hoc Committee on Retroviral-mediated Gene Transfer to Hematopoietic Stem Cells. Available
    • Report from the ASGT Ad Hoc Committee on Retroviral-mediated Gene Transfer to Hematopoietic Stem Cells. Available: http://www.asgt.org/ position§atements/report_042003/index.html.
  • 14
    • 0037497338 scopus 로고    scopus 로고
    • Harmful potential of viral vectors fuels doubts over gene therapy
    • Check E. Harmful potential of viral vectors fuels doubts over gene therapy. Nature 2003; 423: 573-574.
    • (2003) Nature , vol.423 , pp. 573-574
    • Check, E.1
  • 15
    • 2142833382 scopus 로고    scopus 로고
    • Kohn DB, Gansbacher B. Letter to the editors of Nature from the American Society of Gene Therapy (ASGT) and the European Society of Gene Therapy (ESGT). J Gene Med 2003; 5: 641.
    • Kohn DB, Gansbacher B. Letter to the editors of Nature from the American Society of Gene Therapy (ASGT) and the European Society of Gene Therapy (ESGT). J Gene Med 2003; 5: 641.
  • 16
    • 35148848090 scopus 로고    scopus 로고
    • Gene Therapy Advisory Committee 12th Annual Report (pages 10 and 13-15). Available: http://www.advisorybodies.doh.gov.uk/genetics/gtac/ GTAC12thannualReport.pdf.
    • Gene Therapy Advisory Committee 12th Annual Report (pages 10 and 13-15). Available: http://www.advisorybodies.doh.gov.uk/genetics/gtac/ GTAC12thannualReport.pdf.
  • 17
  • 18
    • 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 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: 401-409.
    • (2006) Nat Med , vol.12 , pp. 401-409
    • Ott, M.G.1    Schmidt, M.2    Schwarzwaelder, K.3
  • 19
    • 70349302679 scopus 로고    scopus 로고
    • One of three successfully treated CGD patients in a Swiss-German gene therapy trial died due to his underlying disease: A position statement from the European Society of Gene Therapy (ESGT)
    • European Society of Gene Therapy ESGT
    • European Society of Gene Therapy (ESGT). One of three successfully treated CGD patients in a Swiss-German gene therapy trial died due to his underlying disease: A position statement from the European Society of Gene Therapy (ESGT). J Gene Med 2006; 8: 1435.
    • (2006) J Gene Med , vol.8 , pp. 1435
  • 20
    • 33749624177 scopus 로고    scopus 로고
    • Cancer regression in patients after transfer of genetically engineered lymphocytes
    • Morgan RA, Dudley ME, Wunderlich JR, et al. Cancer regression in patients after transfer of genetically engineered lymphocytes. Science 2006; 314: 126-129.
    • (2006) Science , vol.314 , pp. 126-129
    • Morgan, R.A.1    Dudley, M.E.2    Wunderlich, J.R.3
  • 21
    • 35148836991 scopus 로고    scopus 로고
    • Rush University Medical Center and UCSF researchers found gene therapy appeared to reduce symptoms of Parkinson's disease by 40 percent. Press release from Rush University Medical Center, October 16, 2006; Available
    • Rush University Medical Center and UCSF researchers found gene therapy appeared to reduce symptoms of Parkinson's disease by 40 percent. Press release from Rush University Medical Center, October 16, 2006; Available: http://www.rush.edu/webapps/MEDREL/servlet/NewsRelease?id=802.
  • 22
    • 79955563177 scopus 로고    scopus 로고
    • Subthalamic GAD gene transfer improves brain metabolism associated with clinical recovery in Parkinson's disease
    • Available
    • During MJ, Feigin A, Eidelberg D, et al. Subthalamic GAD gene transfer improves brain metabolism associated with clinical recovery in Parkinson's disease. Abstracts from Neuroscience 2006: The 36th Annual Meeting of the Society for Neuroscience; No. 510.9. Available: http://www.sfn.org/index.cfm?pagename= abstracts_ampublications§ion=publications.
    • Abstracts from Neuroscience 2006: The 36th Annual Meeting of the Society for Neuroscience , Issue.510 .9
    • During, M.J.1    Feigin, A.2    Eidelberg, D.3
  • 23
    • 24944454988 scopus 로고    scopus 로고
    • Current status of gendicine in China: Recombinant human Ad-p53 agent for treatment of cancers
    • Peng Z. Current status of gendicine in China: recombinant human Ad-p53 agent for treatment of cancers. Hum Gene Ther 2005; 16: 1016-10127.
    • (2005) Hum Gene Ther , vol.16 , pp. 1016-10127
    • Peng, Z.1
  • 24
    • 33751562430 scopus 로고    scopus 로고
    • Chinese gene therapy: Splicing out the west?
    • Guo G, Xin H. Chinese gene therapy: splicing out the west? Science 2006; 314: 1232-1235.
    • (2006) Science , vol.314 , pp. 1232-1235
    • Guo, G.1    Xin, H.2
  • 25
    • 35148864348 scopus 로고    scopus 로고
    • NIH Recombinant DNA Advisory Committee RAC, Available
    • NIH Recombinant DNA Advisory Committee (RAC). Available: http://www4.od.nih.gov/oba/rac/aboutrdagt.htm.
  • 26
    • 70349591451 scopus 로고    scopus 로고
    • UK Department of Health:, Available
    • UK Department of Health: Gene Therapy Advisory Committee (GTAC). Available: http://www.doh.gov.uk/genetics/gtac/.
    • Gene Therapy Advisory Committee (GTAC)
  • 29
    • 35148821345 scopus 로고    scopus 로고
    • Gene Therapy Research Advisory Panel (GTRAP) of Australia. Available: http://www.health.gov.au/nhmrc/research/gtrap.htm.
    • Gene Therapy Research Advisory Panel (GTRAP) of Australia. Available: http://www.health.gov.au/nhmrc/research/gtrap.htm.
  • 30
    • 35148874050 scopus 로고    scopus 로고
    • Swiss Expert Committee for Biosafety EFBS/CFSB, trials information. Available
    • Swiss Expert Committee for Biosafety (EFBS/CFSB) gene therapy clinical trials information. Available: http://www.efbs.ch/buwal/eng/fachgebiete/ fg_efbs/rubrik_gentherapie/gen_versuche/index.html.
    • gene therapy clinical
  • 31
    • 35148863867 scopus 로고    scopus 로고
    • Chinese State Food and Drug Administration clinical trials database. Available
    • Chinese State Food and Drug Administration clinical trials database. Available: http://appl.sfda.gov.cn/webportal/ portal.po?UID=DWV1_WOUID_URL_4258.
  • 32
    • 34247327255 scopus 로고    scopus 로고
    • Dutch Ministry of Housing, VROM GGO Office site. Available
    • Dutch Ministry of Housing, Spatial Planning and the Environment (VROM) GGO Office site. Available: http://www.vrom.nl/ggo-vergunningverlening.
    • Spatial Planning and the Environment
  • 33
    • 35148856063 scopus 로고    scopus 로고
    • European Community clinical trials database EudraCT, Available
    • European Community clinical trials database (EudraCT). Available: http://eudract.emea.europa.eu/.
  • 34
    • 0028807727 scopus 로고
    • T lymphocyte-directed gene therapy for ADA-SCID: Initial trial results after 4 years
    • Blaese RM, Culver KW, Miller AD, et al. T lymphocyte-directed gene therapy for ADA-SCID: initial trial results after 4 years. Science 1995; 270: 475-480.
    • (1995) Science , vol.270 , pp. 475-480
    • Blaese, R.M.1    Culver, K.W.2    Miller, A.D.3
  • 35
    • 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, Nagy KZ, Giordano FA, Hotz-Wagenblatt A, Zeller WJ, Fruehauf S. 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 2004; 10: 874-881.
    • (2004) Mol Ther , vol.10 , pp. 874-881
    • Laufs, S.1    Nagy, K.Z.2    Giordano, F.A.3    Hotz-Wagenblatt, A.4    Zeller, W.J.5    Fruehauf, S.6
  • 36
    • 0032545933 scopus 로고    scopus 로고
    • Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans
    • Fire A, Xu S, Montgomery M, Kostas S, Driver S, Mello C. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 1998; 391: 806-811.
    • (1998) Nature , vol.391 , pp. 806-811
    • Fire, A.1    Xu, S.2    Montgomery, M.3    Kostas, S.4    Driver, S.5    Mello, C.6
  • 37
    • 33646891110 scopus 로고    scopus 로고
    • Induction of the interferon response by siRNA is cell type- and duplex length-dependent
    • Reynolds A, Anderson E, Vermeulen A, et al. Induction of the interferon response by siRNA is cell type- and duplex length-dependent. RNA 2006; 12: 988-993.
    • (2006) RNA , vol.12 , pp. 988-993
    • Reynolds, A.1    Anderson, E.2    Vermeulen, A.3
  • 38
    • 35148890145 scopus 로고    scopus 로고
    • Sirna Therapeutics announces programs in strategic research alliance with GlaxoSmithKline. Press release from Sirna Therapeutics. Available: http://phx.corporate-ir.net/phoenix.zhtml?c=141787&p= irol-newsArticle&ID=852037&highlight=.
    • Sirna Therapeutics announces programs in strategic research alliance with GlaxoSmithKline. Press release from Sirna Therapeutics. Available: http://phx.corporate-ir.net/phoenix.zhtml?c=141787&p= irol-newsArticle&ID=852037&highlight=.
  • 39
    • 35148878995 scopus 로고    scopus 로고
    • Merck & Co, Inc. announces completion of acquisition of Sirna Therapeutics, Inc. Press release from Sirna Therapeutics. Available
    • Merck & Co., Inc. announces completion of acquisition of Sirna Therapeutics, Inc. Press release from Sirna Therapeutics. Available: http://phx.corporate-ir.net/phoenix.zhtml?c=141787&p= irol-newsArticle&ID=945592&highlight=.
    • 2&highlight
  • 40
    • 35148889669 scopus 로고    scopus 로고
    • Mirus Bio awarded broad European patent covering intravascular injection method, release from Mirus Bio Corporation. Available
    • Mirus Bio awarded broad European patent covering intravascular injection method. Press release from Mirus Bio Corporation. Available: http://www.mirusbio.com/news/releases/20061031.asp.
    • Press
  • 42
    • 35148901425 scopus 로고    scopus 로고
    • Mirus Bio Corporation enters into agreement with Pfizer, Inc. to evaluate RNAi delivery technology, release from Mirus Bio Corporation. Available
    • Mirus Bio Corporation enters into agreement with Pfizer, Inc. to evaluate RNAi delivery technology. Press release from Mirus Bio Corporation. Available: http://www.mirusbio.com/news/releases/ 20070109.asp.
    • Press
  • 43
    • 27344439351 scopus 로고    scopus 로고
    • Complete and persistent phenotypic correction of phenylketonuria in mice by site-specific genome integration of murine phenylalanine hydroxylase cDNA
    • Chen L, Woo SLC. Complete and persistent phenotypic correction of phenylketonuria in mice by site-specific genome integration of murine phenylalanine hydroxylase cDNA. Proc Natl Acad Sci USA 2005; 102: 15581-15586.
    • (2005) Proc Natl Acad Sci USA , vol.102 , pp. 15581-15586
    • Chen, L.1    Woo, S.L.C.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.