메뉴 건너뛰기




Volumn 20, Issue 5, 2018, Pages

Corrigendum: Gene therapy clinical trials worldwide to 2017: An update (The Journal of Gene Medicine, (2018), 20, 5, (e3015), 10.1002/jgm.3015);Gene therapy clinical trials worldwide to 2017: An update

Author keywords

clinical trials; database; gene therapy; The Journal of Gene Medicine; worldwide

Indexed keywords

ALIPOGENE TIPARVOVEC; AXICABTAGENE CILOLEUCEL; GENDICINE; LUXTURNA; STRIMVELIS; TISAGENLECLEUCEL T; VIRUS VECTOR; VORETIGENE NEPARVOVEC; YESCARTA;

EID: 85048049494     PISSN: 1099498X     EISSN: 15212254     Source Type: Journal    
DOI: 10.1002/jgm.3124     Document Type: Erratum
Times cited : (640)

References (167)
  • 1
    • 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
  • 2
    • 84874346554 scopus 로고    scopus 로고
    • Gene therapy clinical trials worldwide to 2012 – an update
    • Ginn SL, Alexander IE, Edelstein ML, et al. Gene therapy clinical trials worldwide to 2012 – an update. J Gene Med. 2013;15:65-77.
    • (2013) J Gene Med , vol.15 , pp. 65-77
    • Ginn, S.L.1    Alexander, I.E.2    Edelstein, M.L.3
  • 3
    • 35148817461 scopus 로고    scopus 로고
    • Gene therapy clinical trials worldwide to 2007 – an update
    • Edelstein ML, Abedi MR, Wixon J. Gene therapy clinical trials worldwide to 2007 – an update. J Gene Med. 2007;9:833-842.
    • (2007) J Gene Med , vol.9 , pp. 833-842
    • Edelstein, M.L.1    Abedi, M.R.2    Wixon, J.3
  • 4
    • 4444370799 scopus 로고    scopus 로고
    • Gene therapy clinical trials worldwide 1989–2004 – an overview
    • Edelstein ML, Abedi MR, Wixon J, et al. 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
  • 5
    • 80052048463 scopus 로고    scopus 로고
    • Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency
    • 97ra79
    • Gaspar HB, Cooray S, Gilmour KC, et al. Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency. Sci Transl Med. 2011;3. 97ra79
    • (2011) Sci Transl Med , vol.3
    • Gaspar, H.B.1    Cooray, S.2    Gilmour, K.C.3
  • 6
    • 77954833516 scopus 로고    scopus 로고
    • Efficacy of gene therapy for X-linked severe combined immunodeficiency
    • Hacein-Bey-Abina S, Hauer J, Lim A, et al. Efficacy of gene therapy for X-linked severe combined immunodeficiency. N Engl J Med. 2010;363:355-364.
    • (2010) N Engl J Med , vol.363 , pp. 355-364
    • Hacein-Bey-Abina, S.1    Hauer, J.2    Lim, A.3
  • 7
    • 77949438081 scopus 로고    scopus 로고
    • Ten years of gene therapy for primary immune deficiencies. Hematology 2009 – the Education Program of the American Society of Hematology
    • Aiuti A, Roncarolo MG. Ten years of gene therapy for primary immune deficiencies. Hematology 2009 – the Education Program of the American Society of Hematology. Education Program. 2009;2009:682-689.
    • (2009) Education Program , vol.2009 , pp. 682-689
    • Aiuti, A.1    Roncarolo, M.G.2
  • 8
    • 80052050274 scopus 로고    scopus 로고
    • Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction
    • 97ra80
    • Gaspar HB, Cooray S, Gilmour KC, et al. Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction. Sci Transl Med. 2011;3. 97ra80
    • (2011) Sci Transl Med , vol.3
    • Gaspar, H.B.1    Cooray, S.2    Gilmour, K.C.3
  • 9
    • 77956928344 scopus 로고    scopus 로고
    • Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia
    • Cavazzana-Calvo M, Payen E, Negre O, et al. Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia. Nature. 2010;467:318-322.
    • (2010) Nature , vol.467 , pp. 318-322
    • Cavazzana-Calvo, M.1    Payen, E.2    Negre, O.3
  • 10
    • 78149482538 scopus 로고    scopus 로고
    • Stem-cell gene therapy for the Wiskott-Aldrich syndrome
    • Boztug K, Schmidt M, Schwarzer A, et al. Stem-cell gene therapy for the Wiskott-Aldrich syndrome. N Engl J Med. 2010;363:1918-1927.
    • (2010) N Engl J Med , vol.363 , pp. 1918-1927
    • Boztug, K.1    Schmidt, M.2    Schwarzer, A.3
  • 11
    • 44249120315 scopus 로고    scopus 로고
    • Effect of gene therapy on visual function in Leber's congenital amaurosis
    • Bainbridge JW, Smith AJ, Barker SS, et al. Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med. 2008;358:2231-2239.
    • (2008) N Engl J Med , vol.358 , pp. 2231-2239
    • Bainbridge, J.W.1    Smith, A.J.2    Barker, S.S.3
  • 12
    • 44249085878 scopus 로고    scopus 로고
    • Safety and efficacy of gene transfer for Leber's congenital amaurosis
    • Maguire AM, Simonelli F, Pierce EA, et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med. 2008;358:2240-2248.
    • (2008) N Engl J Med , vol.358 , pp. 2240-2248
    • Maguire, A.M.1    Simonelli, F.2    Pierce, E.A.3
  • 13
    • 70350620424 scopus 로고    scopus 로고
    • Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
    • Maguire AM, High KA, Auricchio A, et al. Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet. 2009;374:1597-1605.
    • (2009) Lancet , vol.374 , pp. 1597-1605
    • Maguire, A.M.1    High, K.A.2    Auricchio, A.3
  • 14
    • 84855161388 scopus 로고    scopus 로고
    • Adenovirus-associated virus vector-mediated gene transfer in hemophilia B
    • Nathwani AC, Tuddenham EG, Rangarajan S, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med. 2011;365:2357-2365.
    • (2011) N Engl J Med , vol.365 , pp. 2357-2365
    • Nathwani, A.C.1    Tuddenham, E.G.2    Rangarajan, S.3
  • 15
    • 70449427834 scopus 로고    scopus 로고
    • Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy
    • Cartier N, Hacein-Bey-Abina S, Bartholomae CC, et al. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science. 2009;326:818-823.
    • (2009) Science , vol.326 , pp. 818-823
    • Cartier, N.1    Hacein-Bey-Abina, S.2    Bartholomae, C.C.3
  • 16
    • 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
  • 17
    • 79952304769 scopus 로고    scopus 로고
    • Tumor regression in patients with metastatic synovial cell sarcoma and melanoma using genetically engineered lymphocytes reactive with NY-ESO-1
    • Robbins PF, Morgan RA, Feldman SA, et al. Tumor regression in patients with metastatic synovial cell sarcoma and melanoma using genetically engineered lymphocytes reactive with NY-ESO-1. J Clin Oncol. 2011;29:917-924.
    • (2011) J Clin Oncol , vol.29 , pp. 917-924
    • Robbins, P.F.1    Morgan, R.A.2    Feldman, S.A.3
  • 18
    • 80051775476 scopus 로고    scopus 로고
    • T cells with chimeric antigen receptors have potent antitumor effects and can establish memory in patients with advanced leukemia
    • 95ra73
    • Kalos M, Levine BL, Porter DL, et al. T cells with chimeric antigen receptors have potent antitumor effects and can establish memory in patients with advanced leukemia. Sci Transl Med. 2011;3. 95ra73
    • (2011) Sci Transl Med , vol.3
    • Kalos, M.1    Levine, B.L.2    Porter, D.L.3
  • 19
    • 80051720194 scopus 로고    scopus 로고
    • Chimeric antigen receptor-modified T cells in chronic lymphoid leukemia
    • Porter DL, Levine BL, Kalos M, et al. Chimeric antigen receptor-modified T cells in chronic lymphoid leukemia. N Engl J Med. 2011;365:725-733.
    • (2011) N Engl J Med , vol.365 , pp. 725-733
    • Porter, D.L.1    Levine, B.L.2    Kalos, M.3
  • 20
    • 84941686285 scopus 로고    scopus 로고
    • CAR therapy: the CD19 paradigm
    • Sadelain M. CAR therapy: the CD19 paradigm. J Clin Invest. 2015;125:3392-3400.
    • (2015) J Clin Invest , vol.125 , pp. 3392-3400
    • Sadelain, M.1
  • 22
    • 84908073316 scopus 로고    scopus 로고
    • Chimeric antigen receptor T cells for sustained remissions in leukemia
    • Maude SL, Frey N, Shaw PA, et al. Chimeric antigen receptor T cells for sustained remissions in leukemia. N Engl J Med. 2014;371:1507-1517.
    • (2014) N Engl J Med , vol.371 , pp. 1507-1517
    • Maude, S.L.1    Frey, N.2    Shaw, P.A.3
  • 23
    • 84896335556 scopus 로고    scopus 로고
    • Efficacy and toxicity management of 19-28z CAR T cell therapy in B cell acute lymphoblastic leukemia
    • 224ra225
    • Davila ML, Riviere I, Wang X, et al. Efficacy and toxicity management of 19-28z CAR T cell therapy in B cell acute lymphoblastic leukemia. Sci Transl Med. 2014;6. 224ra225
    • (2014) Sci Transl Med , vol.6
    • Davila, M.L.1    Riviere, I.2    Wang, X.3
  • 24
    • 84876325876 scopus 로고    scopus 로고
    • Chimeric antigen receptor-modified T cells for acute lymphoid leukemia
    • Grupp SA, Kalos M, Barrett D, et al. Chimeric antigen receptor-modified T cells for acute lymphoid leukemia. N Engl J Med. 2013;368:1509-1518.
    • (2013) N Engl J Med , vol.368 , pp. 1509-1518
    • Grupp, S.A.1    Kalos, M.2    Barrett, D.3
  • 25
    • 84876005284 scopus 로고    scopus 로고
    • CD19-targeted T cells rapidly induce molecular remissions in adults with chemotherapy-refractory acute lymphoblastic leukemia
    • 177ra138
    • Brentjens RJ, Davila ML, Riviere I, et al. CD19-targeted T cells rapidly induce molecular remissions in adults with chemotherapy-refractory acute lymphoblastic leukemia. Sci Transl Med. 2013;5. 177ra138
    • (2013) Sci Transl Med , vol.5
    • Brentjens, R.J.1    Davila, M.L.2    Riviere, I.3
  • 26
    • 85018686063 scopus 로고    scopus 로고
    • Chimeric antigen receptors: a cell and gene therapy perspective
    • Riviere I, Sadelain M. Chimeric antigen receptors: a cell and gene therapy perspective. Mol Ther. 2017;25:1117-1124.
    • (2017) Mol Ther , vol.25 , pp. 1117-1124
    • Riviere, I.1    Sadelain, M.2
  • 27
    • 85018795014 scopus 로고    scopus 로고
    • Phase I escalating-dose trial of CAR-T therapy targeting CEA+ metastatic colorectal cancers
    • Zhang C, Wang Z, Yang Z, et al. Phase I escalating-dose trial of CAR-T therapy targeting CEA+ metastatic colorectal cancers. Mol Ther. 2017;25:1248-1258.
    • (2017) Mol Ther , vol.25 , pp. 1248-1258
    • Zhang, C.1    Wang, Z.2    Yang, Z.3
  • 28
    • 84890814629 scopus 로고    scopus 로고
    • Breakthrough of the year 2013. Cancer immunotherapy
    • Couzin-Frankel J. Breakthrough of the year 2013. Cancer immunotherapy. Science. 2013;342:1432-1433.
    • (2013) Science , vol.342 , pp. 1432-1433
    • Couzin-Frankel, J.1
  • 29
    • 85048030872 scopus 로고    scopus 로고
    • [7 August
    • Emily Whitehead Foundation. Celebrating 5 Years Cancer Free! 2017. http://emilywhiteheadfoundation.org/celebrating-5-years-cancer-free/ [7 August 2017].
    • (2017) Celebrating 5 Years Cancer Free! 2017
  • 30
    • 85048038644 scopus 로고    scopus 로고
    • FDA Approval Brings First Gene Therapy to the United States
    • US Food and Drug Administration Newsroom Website Press Release 30 August, [3 April 2018]
    • US Food and Drug Administration. FDA Approval Brings First Gene Therapy to the United States. CAR T-Cell Therapy Approved to Treat Certain Children and Young Adults with B-Cell Acute Lymphoblastic Leukemia. US Food and Drug Administration Newsroom Website: Press Release 30 August 2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm574058.htm [3 April 2018].
    • (2017) CAR T-Cell Therapy Approved to Treat Certain Children and Young Adults with B-Cell Acute Lymphoblastic Leukemia.
  • 31
    • 85021976256 scopus 로고    scopus 로고
    • HER2-specific chimeric antigen receptor-modified virus-specific T cells for progressive glioblastoma: a phase 1 dose-escalation trial
    • Ahmed N, Brawley V, Hegde M, et al. HER2-specific chimeric antigen receptor-modified virus-specific T cells for progressive glioblastoma: a phase 1 dose-escalation trial. JAMA Oncol. 2017;3:1094-1101.
    • (2017) JAMA Oncol , vol.3 , pp. 1094-1101
    • Ahmed, N.1    Brawley, V.2    Hegde, M.3
  • 32
    • 85007288271 scopus 로고    scopus 로고
    • Regression of glioblastoma after chimeric antigen receptor T-cell therapy
    • Brown CE, Alizadeh D, Starr R, et al. Regression of glioblastoma after chimeric antigen receptor T-cell therapy. N Engl J Med. 2016;375:2561-2569.
    • (2016) N Engl J Med , vol.375 , pp. 2561-2569
    • Brown, C.E.1    Alizadeh, D.2    Starr, R.3
  • 33
    • 85025480254 scopus 로고    scopus 로고
    • A single dose of peripherally infused EGFRvIII-directed CAR T cells mediates antigen loss and induces adaptive resistance in patients with recurrent glioblastoma
    • eaaa0984
    • O'Rourke DM, Nasrallah MP, Desai A, et al. A single dose of peripherally infused EGFRvIII-directed CAR T cells mediates antigen loss and induces adaptive resistance in patients with recurrent glioblastoma. Sci Transl Med. 2017;9. eaaa0984
    • (2017) Sci Transl Med , vol.9
    • O'Rourke, D.M.1    Nasrallah, M.P.2    Desai, A.3
  • 34
    • 84941179388 scopus 로고    scopus 로고
    • Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial
    • Alton EW, Armstrong DK, Ashby D, et al. Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial. Lancet Respir Med. 2015;3:684-691.
    • (2015) Lancet Respir Med , vol.3 , pp. 684-691
    • Alton, E.W.1    Armstrong, D.K.2    Ashby, D.3
  • 35
    • 85018781253 scopus 로고    scopus 로고
    • Evolving gene therapy in primary immunodeficiency
    • Thrasher AJ, Williams DA. Evolving gene therapy in primary immunodeficiency. Mol Ther. 2017;25:1132-1141.
    • (2017) Mol Ther , vol.25 , pp. 1132-1141
    • Thrasher, A.J.1    Williams, D.A.2
  • 36
    • 85006208770 scopus 로고    scopus 로고
    • Clinical manufacturing of CAR T cells: foundation of a promising therapy
    • 16015
    • Wang X, Riviere I. Clinical manufacturing of CAR T cells: foundation of a promising therapy. Mol Ther Oncolytics. 2016;3. 16015
    • (2016) Mol Ther Oncolytics , vol.3
    • Wang, X.1    Riviere, I.2
  • 37
    • 0028789792 scopus 로고
    • Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients
    • Bordignon C, Notarangelo LD, Nobili N, et al. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients. Science. 1995;270:470-475.
    • (1995) Science , vol.270 , pp. 470-475
    • Bordignon, C.1    Notarangelo, L.D.2    Nobili, N.3
  • 38
    • 85017415923 scopus 로고    scopus 로고
    • Gene therapy for ADA-SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products
    • Aiuti A, Roncarolo MG, Naldini L. Gene therapy for ADA-SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products. EMBO Mol Med. 2017;9:737-740.
    • (2017) EMBO Mol Med , vol.9 , pp. 737-740
    • Aiuti, A.1    Roncarolo, M.G.2    Naldini, L.3
  • 39
    • 84975701789 scopus 로고    scopus 로고
    • ADA-SCID gene therapy endorsed by European Medicines Agency for marketing authorization
    • Yla-Herttuala S. ADA-SCID gene therapy endorsed by European Medicines Agency for marketing authorization. Mol Ther. 2016;24:1013-1014.
    • (2016) Mol Ther , vol.24 , pp. 1013-1014
    • Yla-Herttuala, S.1
  • 40
    • 84895427883 scopus 로고    scopus 로고
    • Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency
    • Carbonaro DA, Zhang L, Jin X, et al. Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency. Mol Ther. 2014;22:607-622.
    • (2014) Mol Ther , vol.22 , pp. 607-622
    • Carbonaro, D.A.1    Zhang, L.2    Jin, X.3
  • 41
    • 84897550064 scopus 로고    scopus 로고
    • Gene therapy for Wiskott-Aldrich syndrome – long-term efficacy and genotoxicity
    • 227ra233
    • Braun CJ, Boztug K, Paruzynski A, et al. Gene therapy for Wiskott-Aldrich syndrome – long-term efficacy and genotoxicity. Sci Transl Med. 2014;6. 227ra233
    • (2014) Sci Transl Med , vol.6
    • Braun, C.J.1    Boztug, K.2    Paruzynski, A.3
  • 42
    • 84928389399 scopus 로고    scopus 로고
    • Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome
    • Hacein-Bey Abina S, Gaspar HB, Blondeau J, et al. Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome. JAMA. 2015;313:1550-1563.
    • (2015) JAMA , vol.313 , pp. 1550-1563
    • Hacein-Bey Abina, S.1    Gaspar, H.B.2    Blondeau, J.3
  • 43
    • 84964514054 scopus 로고    scopus 로고
    • Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency
    • 335ra357
    • De Ravin SS, Wu X, Moir S, et al. Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency. Sci Transl Med. 2016;8. 335ra357
    • (2016) Sci Transl Med , vol.8
    • De Ravin, S.S.1    Wu, X.2    Moir, S.3
  • 44
    • 85029504375 scopus 로고    scopus 로고
    • Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult
    • Morris EC, Fox T, Chakraverty R, et al. Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult. Blood. 2017;130:1327-1335.
    • (2017) Blood , vol.130 , pp. 1327-1335
    • Morris, E.C.1    Fox, T.2    Chakraverty, R.3
  • 45
    • 84879873039 scopus 로고    scopus 로고
    • Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy
    • 1233158
    • Biffi A, Montini E, Lorioli L, et al. Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science. 2013;341. 1233158
    • (2013) Science , vol.341
    • Biffi, A.1    Montini, E.2    Lorioli, L.3
  • 46
    • 85017353385 scopus 로고    scopus 로고
    • Hematopoietic stem cell gene therapy for storage disease: current and new indications
    • Biffi A. Hematopoietic stem cell gene therapy for storage disease: current and new indications. Mol Ther. 2017;25:1155-1162.
    • (2017) Mol Ther , vol.25 , pp. 1155-1162
    • Biffi, A.1
  • 47
    • 85032463285 scopus 로고    scopus 로고
    • Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy
    • Eichler F, Duncan C, Musolino PL, et al. Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy. N Engl J Med. 2017;377:1630-1638.
    • (2017) N Engl J Med , vol.377 , pp. 1630-1638
    • Eichler, F.1    Duncan, C.2    Musolino, P.L.3
  • 48
    • 85014855434 scopus 로고    scopus 로고
    • Gene therapy in a patient with sickle cell disease
    • Ribeil JA, Hacein-Bey-Abina S, Payen E, et al. Gene therapy in a patient with sickle cell disease. N Engl J Med. 2017;376:848-855.
    • (2017) N Engl J Med , vol.376 , pp. 848-855
    • Ribeil, J.A.1    Hacein-Bey-Abina, S.2    Payen, E.3
  • 49
    • 84958951097 scopus 로고    scopus 로고
    • Gene therapy of the beta-hemoglobinopathies by lentiviral transfer of the beta(A(T87Q))-globin gene
    • Negre O, Eggimann AV, Beuzard Y, et al. Gene therapy of the beta-hemoglobinopathies by lentiviral transfer of the beta(A(T87Q))-globin gene. Hum Gene Ther. 2016;27:148-165.
    • (2016) Hum Gene Ther , vol.27 , pp. 148-165
    • Negre, O.1    Eggimann, A.V.2    Beuzard, Y.3
  • 50
    • 85016445049 scopus 로고    scopus 로고
    • Gene therapy for beta-hemoglobinopathies
    • Cavazzana M, Antoniani C, Miccio A. Gene therapy for beta-hemoglobinopathies. Mol Ther. 2017;25:1142-1154.
    • (2017) Mol Ther , vol.25 , pp. 1142-1154
    • Cavazzana, M.1    Antoniani, C.2    Miccio, A.3
  • 51
    • 85017403198 scopus 로고    scopus 로고
    • 5 Year expression and neutrophil defect repair after gene therapy in alpha-1 antitrypsin deficiency
    • Mueller C, Gernoux G, Gruntman AM, et al. 5 Year expression and neutrophil defect repair after gene therapy in alpha-1 antitrypsin deficiency. Mol Ther. 2017;25:1387-1394.
    • (2017) Mol Ther , vol.25 , pp. 1387-1394
    • Mueller, C.1    Gernoux, G.2    Gruntman, A.M.3
  • 52
    • 85048031119 scopus 로고    scopus 로고
    • Preliminary results from a phase 1/2 dose escalation trial of an investigational AAV-mediated gene therapy for hemophilia A
    • Online Conference Proceedings; 9 December, [30 January 2018]
    • George LA, Ragni MV, Samelson-Jones BJ, et al. Preliminary results from a phase 1/2 dose escalation trial of an investigational AAV-mediated gene therapy for hemophilia A. American Society of Hematology 59th Annual Meeting and Exposition. Online Conference Proceedings; 9 December 2017. https://ash.confex.com/ash/2017/webprogram/Paper108679.html [30 January 2018].
    • (2017) American Society of Hematology 59th Annual Meeting and Exposition.
    • George, L.A.1    Ragni, M.V.2    Samelson-Jones, B.J.3
  • 53
    • 84911383748 scopus 로고    scopus 로고
    • Long-term safety and efficacy of factor IX gene therapy in hemophilia B
    • Nathwani AC, Reiss UM, Tuddenham EG, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med. 2014;371:1994-2004.
    • (2014) N Engl J Med , vol.371 , pp. 1994-2004
    • Nathwani, A.C.1    Reiss, U.M.2    Tuddenham, E.G.3
  • 55
    • 85038006730 scopus 로고    scopus 로고
    • Hemophilia B gene therapy with a high-specific-activity factor IX variant
    • George LA, Sullivan SK, Giermasz A, et al. Hemophilia B gene therapy with a high-specific-activity factor IX variant. N Engl J Med. 2017;377:2215-2227.
    • (2017) N Engl J Med , vol.377 , pp. 2215-2227
    • George, L.A.1    Sullivan, S.K.2    Giermasz, A.3
  • 56
    • 85017180555 scopus 로고    scopus 로고
    • Taking stock of retinal gene therapy: looking back and moving forward
    • Bennett J. Taking stock of retinal gene therapy: looking back and moving forward. Mol Ther. 2017;25:1076-1094.
    • (2017) Mol Ther , vol.25 , pp. 1076-1094
    • Bennett, J.1
  • 57
    • 84991408036 scopus 로고    scopus 로고
    • Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial
    • Bennett J, Wellman J, Marshall KA, et al. Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial. Lancet. 2016;388:661-672.
    • (2016) Lancet , vol.388 , pp. 661-672
    • Bennett, J.1    Wellman, J.2    Marshall, K.A.3
  • 58
    • 85003977057 scopus 로고    scopus 로고
    • Phase 2a randomized clinical trial: safety and post hoc analysis of subretinal rAAV.sFLT-1 for wet age-related macular degeneration. E
    • Constable IJ, Pierce CM, Lai CM, et al. Phase 2a randomized clinical trial: safety and post hoc analysis of subretinal rAAV.sFLT-1 for wet age-related macular degeneration. E. Biol Med. 2016;14:168-175.
    • (2016) Biol Med , vol.14 , pp. 168-175
    • Constable, I.J.1    Pierce, C.M.2    Lai, C.M.3
  • 59
    • 84952639507 scopus 로고    scopus 로고
    • Gene therapy with recombinant adeno-associated vectors for neovascular age-related macular degeneration: 1 year follow-up of a phase 1 randomised clinical trial
    • Rakoczy EP, Lai CM, Magno AL, et al. Gene therapy with recombinant adeno-associated vectors for neovascular age-related macular degeneration: 1 year follow-up of a phase 1 randomised clinical trial. Lancet. 2015;386:2395-2403.
    • (2015) Lancet , vol.386 , pp. 2395-2403
    • Rakoczy, E.P.1    Lai, C.M.2    Magno, A.L.3
  • 60
    • 85044126094 scopus 로고    scopus 로고
    • US Food and Drug Administration Newsroom Website Press Release 19 December, [3 April 2018]
    • US Food and Drug Administration. FDA Approves Novel Gene Therapy to Treat Patients with a Rare Form of Inherited Vision Loss. US Food and Drug Administration Newsroom Website: Press Release 19 December 2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm589467.htm [3 April 2018].
    • (2017) FDA Approves Novel Gene Therapy to Treat Patients with a Rare Form of Inherited Vision Loss
  • 62
    • 85032715202 scopus 로고    scopus 로고
    • Single-dose gene-replacement therapy for spinal muscular atrophy
    • Mendell JR, Al-Zaidy S, Shell R, et al. Single-dose gene-replacement therapy for spinal muscular atrophy. N Engl J Med. 2017;377:1713-1722.
    • (2017) N Engl J Med , vol.377 , pp. 1713-1722
    • Mendell, J.R.1    Al-Zaidy, S.2    Shell, R.3
  • 64
    • 85040170618 scopus 로고    scopus 로고
    • Axicabtagene ciloleucel CAR T-cell therapy in refractory large B-cell lymphoma
    • Neelapu SS, Locke FL, Bartlett NL, et al. Axicabtagene ciloleucel CAR T-cell therapy in refractory large B-cell lymphoma. N Engl J Med. 2017;377:2531-2544.
    • (2017) N Engl J Med , vol.377 , pp. 2531-2544
    • Neelapu, S.S.1    Locke, F.L.2    Bartlett, N.L.3
  • 65
    • 84960984035 scopus 로고    scopus 로고
    • Manufacturing of recombinant adeno-associated viral vectors for clinical trials
    • Clement N, Grieger JC. Manufacturing of recombinant adeno-associated viral vectors for clinical trials. Mol Ther Methods Clin Dev. 2016;3. 16002
    • (2016) Mol Ther Methods Clin Dev , vol.3 , pp. 16002
    • Clement, N.1    Grieger, J.C.2
  • 66
    • 85020105413 scopus 로고    scopus 로고
    • Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects
    • Baruteau J, Waddington SN, Alexander IE, et al. Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects. J Inherit Metab Dis. 2017;40:497-517.
    • (2017) J Inherit Metab Dis , vol.40 , pp. 497-517
    • Baruteau, J.1    Waddington, S.N.2    Alexander, I.E.3
  • 67
    • 0024328536 scopus 로고
    • Altering the genome by homologous recombination
    • Capecchi MR. Altering the genome by homologous recombination. Science. 1989;244:1288-1292.
    • (1989) Science , vol.244 , pp. 1288-1292
    • Capecchi, M.R.1
  • 68
    • 0031897270 scopus 로고    scopus 로고
    • Human gene targeting by viral vectors
    • Russell DW, Hirata RK. Human gene targeting by viral vectors. Nat Genet. 1998;18:325-330.
    • (1998) Nat Genet , vol.18 , pp. 325-330
    • Russell, D.W.1    Hirata, R.K.2
  • 69
    • 84923138903 scopus 로고    scopus 로고
    • Promoterless gene targeting without nucleases ameliorates haemophilia B in mice
    • Barzel A, Paulk NK, Shi Y, et al. Promoterless gene targeting without nucleases ameliorates haemophilia B in mice. Nature. 2015;517:360-364.
    • (2015) Nature , vol.517 , pp. 360-364
    • Barzel, A.1    Paulk, N.K.2    Shi, Y.3
  • 70
    • 84943601842 scopus 로고    scopus 로고
    • In vivo genome editing of the albumin locus as a platform for protein replacement therapy
    • Sharma R, Anguela XM, Doyon Y, et al. In vivo genome editing of the albumin locus as a platform for protein replacement therapy. Blood. 2015;126:1777-1784.
    • (2015) Blood , vol.126 , pp. 1777-1784
    • Sharma, R.1    Anguela, X.M.2    Doyon, Y.3
  • 71
    • 85048035903 scopus 로고    scopus 로고
    • Targeted exon skipping to correct exon duplications in the dystrophin gene
    • Greer KL, Lochmuller H, Flanigan K, et al. Targeted exon skipping to correct exon duplications in the dystrophin gene. Mol Ther Nucleic Acids. 2014;e155:3.
    • (2014) Mol Ther Nucleic Acids , vol.e155 , pp. 3
    • Greer, K.L.1    Lochmuller, H.2    Flanigan, K.3
  • 72
    • 69949107887 scopus 로고    scopus 로고
    • Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study
    • Kinali M, Arechavala-Gomeza V, Feng L, et al. Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study. Lancet Neurol. 2009;8:918-928.
    • (2009) Lancet Neurol , vol.8 , pp. 918-928
    • Kinali, M.1    Arechavala-Gomeza, V.2    Feng, L.3
  • 73
    • 84941079088 scopus 로고    scopus 로고
    • Special issue: Gene therapy with emphasis on RNA interference
    • Lundstrom K. Special issue: Gene therapy with emphasis on RNA interference. Virus. 2015;7:4482-4487.
    • (2015) Virus , vol.7 , pp. 4482-4487
    • Lundstrom, K.1
  • 74
    • 84898755357 scopus 로고    scopus 로고
    • RNA interference gene therapy in dominant retinitis pigmentosa and cone-rod dystrophy mouse models caused by GCAP1 mutations
    • Jiang L, Frederick JM, Baehr W. RNA interference gene therapy in dominant retinitis pigmentosa and cone-rod dystrophy mouse models caused by GCAP1 mutations. Front Mol Neurosci. 2014;7:25.
    • (2014) Front Mol Neurosci , vol.7 , pp. 25
    • Jiang, L.1    Frederick, J.M.2    Baehr, W.3
  • 75
    • 0032545933 scopus 로고    scopus 로고
    • Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans
    • Fire A, Xu S, Montgomery MK, et al. 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.K.3
  • 76
    • 84877120847 scopus 로고    scopus 로고
    • First-in-humans trial of an RNA interference therapeutic targeting VEGF and KSP in cancer patients with liver involvement
    • Tabernero J, Shapiro GI, LoRusso PM, et al. First-in-humans trial of an RNA interference therapeutic targeting VEGF and KSP in cancer patients with liver involvement. Cancer Discov. 2013;3:406-417.
    • (2013) Cancer Discov , vol.3 , pp. 406-417
    • Tabernero, J.1    Shapiro, G.I.2    LoRusso, P.M.3
  • 77
    • 84986552471 scopus 로고    scopus 로고
    • RNA-targeted therapeutics in cancer clinical trials: current status and future directions
    • Barata P, Sood AK, Hong DS. RNA-targeted therapeutics in cancer clinical trials: current status and future directions. Cancer Treat Rev. 2016;50:35-47.
    • (2016) Cancer Treat Rev , vol.50 , pp. 35-47
    • Barata, P.1    Sood, A.K.2    Hong, D.S.3
  • 78
    • 85017268987 scopus 로고    scopus 로고
    • Refining strategies to translate genome editing to the clinic
    • Cornu TI, Mussolino C, Cathomen T. Refining strategies to translate genome editing to the clinic. Nat Med. 2017;23:415-423.
    • (2017) Nat Med , vol.23 , pp. 415-423
    • Cornu, T.I.1    Mussolino, C.2    Cathomen, T.3
  • 79
    • 33750094787 scopus 로고    scopus 로고
    • Nobel Prize in Physiology or Medicine. Method to silence genes earns loud praise
    • Couzin J. Nobel Prize in Physiology or Medicine. Method to silence genes earns loud praise. Science. 2006;314:34.
    • (2006) Science , vol.314 , pp. 34
    • Couzin, J.1
  • 80
    • 80051690306 scopus 로고    scopus 로고
    • Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study
    • Cirak S, Arechavala-Gomeza V, Guglieri M, et al. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study. Lancet. 2011;378:595-605.
    • (2011) Lancet , vol.378 , pp. 595-605
    • Cirak, S.1    Arechavala-Gomeza, V.2    Guglieri, M.3
  • 81
    • 84907991204 scopus 로고    scopus 로고
    • Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study
    • Voit T, Topaloglu H, Straub V, et al. Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study. Lancet Neurol. 2014;13:987-996.
    • (2014) Lancet Neurol , vol.13 , pp. 987-996
    • Voit, T.1    Topaloglu, H.2    Straub, V.3
  • 82
    • 85011728863 scopus 로고    scopus 로고
    • Progress and prospects of gene therapy clinical trials for the muscular dystrophies
    • Bengtsson NE, Seto JT, Hall JK, et al. Progress and prospects of gene therapy clinical trials for the muscular dystrophies. Hum Mol Genet. 2016;25:R9-R17.
    • (2016) Hum Mol Genet , vol.25 , pp. R9-R17
    • Bengtsson, N.E.1    Seto, J.T.2    Hall, J.K.3
  • 83
    • 85032714516 scopus 로고    scopus 로고
    • Nusinersen versus sham control in infantile-onset spinal muscular atrophy
    • Finkel RS, Mercuri E, Darras BT, et al. Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N Engl J Med. 2017;377:1723-1732.
    • (2017) N Engl J Med , vol.377 , pp. 1723-1732
    • Finkel, R.S.1    Mercuri, E.2    Darras, B.T.3
  • 84
    • 85017446297 scopus 로고    scopus 로고
    • Progress toward gene therapy for Duchenne muscular dystrophy
    • Chamberlain JR, Chamberlain JS. Progress toward gene therapy for Duchenne muscular dystrophy. Mol Ther. 2017;25:1125-1131.
    • (2017) Mol Ther , vol.25 , pp. 1125-1131
    • Chamberlain, J.R.1    Chamberlain, J.S.2
  • 85
    • 84995595264 scopus 로고    scopus 로고
    • US Food and Drug Administration Newsroom Website Press Release 19 September, [30 January 2018]
    • US Food and Drug Administration. FDA Grants Accelerated Approval to First Drug for Duchenne Muscular Dystrophy. US Food and Drug Administration Newsroom Website: Press Release 19 September 2016; https://www.fda.gov/newsevents/newsroom/pressannouncements/ucm521263.htm [30 January 2018].
    • (2016) FDA Grants Accelerated Approval to First Drug for Duchenne Muscular Dystrophy
  • 86
    • 85011409933 scopus 로고    scopus 로고
    • approves eteplirsen for Duchenne muscular dystrophy: the next chapter in the eteplirsen saga
    • Aartsma-Rus A, Krieg AMFDA. approves eteplirsen for Duchenne muscular dystrophy: the next chapter in the eteplirsen saga. Nucleic Acid Ther. 2017;27:1-3.
    • (2017) Nucleic Acid Ther , vol.27 , pp. 1-3
    • Aartsma-Rus, A.1    Krieg, A.M.F.D.A.2
  • 87
    • 85016512565 scopus 로고    scopus 로고
    • FDA Approval of nusinersen for spinal muscular atrophy makes 2016 the year of splice modulating oligonucleotides
    • Aartsma-Rus A. FDA Approval of nusinersen for spinal muscular atrophy makes 2016 the year of splice modulating oligonucleotides. Nucleic Acid Ther. 2017;27:67-69.
    • (2017) Nucleic Acid Ther , vol.27 , pp. 67-69
    • Aartsma-Rus, A.1
  • 88
    • 72149090954 scopus 로고    scopus 로고
    • A simple cipher governs DNA recognition by TAL effectors
    • Moscou MJ, Bogdanove AJ. A simple cipher governs DNA recognition by TAL effectors. Science. 2009;326:1501.
    • (2009) Science , vol.326 , pp. 1501
    • Moscou, M.J.1    Bogdanove, A.J.2
  • 89
    • 72149110399 scopus 로고    scopus 로고
    • Breaking the code of DNA binding specificity of TAL-type III effectors
    • Boch J, Scholze H, Schornack S, et al. Breaking the code of DNA binding specificity of TAL-type III effectors. Science. 2009;326:1509-1512.
    • (2009) Science , vol.326 , pp. 1509-1512
    • Boch, J.1    Scholze, H.2    Schornack, S.3
  • 90
    • 0032437591 scopus 로고    scopus 로고
    • Toward controlling gene expression at will: specific regulation of the erbB-2/HER-2 promoter by using polydactyl zinc finger proteins constructed from modular building blocks
    • Beerli RR, Segal DJ, Dreier B, et al. Toward controlling gene expression at will: specific regulation of the erbB-2/HER-2 promoter by using polydactyl zinc finger proteins constructed from modular building blocks. Proc Natl Acad Sci U S A. 1998;95:14628-14633.
    • (1998) Proc Natl Acad Sci U S A , vol.95 , pp. 14628-14633
    • Beerli, R.R.1    Segal, D.J.2    Dreier, B.3
  • 91
    • 0030032063 scopus 로고    scopus 로고
    • Hybrid restriction enzymes: zinc finger fusions to Fok I cleavage domain
    • Kim YG, Cha J, Chandrasegaran S. Hybrid restriction enzymes: zinc finger fusions to Fok I cleavage domain. Proc Natl Acad Sci U S A. 1996;93:1156-1160.
    • (1996) Proc Natl Acad Sci U S A , vol.93 , pp. 1156-1160
    • Kim, Y.G.1    Cha, J.2    Chandrasegaran, S.3
  • 92
    • 84856479947 scopus 로고    scopus 로고
    • Gene-editing nucleases
    • Baker M. Gene-editing nucleases. Nat Methods. 2012;9:23-26.
    • (2012) Nat Methods , vol.9 , pp. 23-26
    • Baker, M.1
  • 93
    • 84895487305 scopus 로고    scopus 로고
    • Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV
    • Tebas P, Stein D, Tang WW, et al. Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV. N Engl J Med. 2014;370:901-910.
    • (2014) N Engl J Med , vol.370 , pp. 901-910
    • Tebas, P.1    Stein, D.2    Tang, W.W.3
  • 94
    • 84946925920 scopus 로고    scopus 로고
    • Leukaemia success heralds wave of gene-editing therapies
    • Reardon S. Leukaemia success heralds wave of gene-editing therapies. Nature. 2015;527:146-147.
    • (2015) Nature , vol.527 , pp. 146-147
    • Reardon, S.1
  • 95
    • 85011392403 scopus 로고    scopus 로고
    • Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells
    • eaaj2013
    • Qasim W, Zhan H, Samarasinghe S, et al. Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells. Sci Transl Med. 2017;9. eaaj2013
    • (2017) Sci Transl Med , vol.9
    • Qasim, W.1    Zhan, H.2    Samarasinghe, S.3
  • 96
    • 84865070369 scopus 로고    scopus 로고
    • A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity
    • Jinek M, Chylinski K, Fonfara I, et al. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science. 2012;337:816-821.
    • (2012) Science , vol.337 , pp. 816-821
    • Jinek, M.1    Chylinski, K.2    Fonfara, I.3
  • 97
    • 84873729095 scopus 로고    scopus 로고
    • Multiplex genome engineering using CRISPR/Cas systems
    • Cong L, Ran FA, Cox D, et al. Multiplex genome engineering using CRISPR/Cas systems. Science. 2013;339:819-823.
    • (2013) Science , vol.339 , pp. 819-823
    • Cong, L.1    Ran, F.A.2    Cox, D.3
  • 98
    • 84873734105 scopus 로고    scopus 로고
    • RNA-guided human genome engineering via Cas9
    • Mali P, Yang L, Esvelt KM, et al. RNA-guided human genome engineering via Cas9. Science. 2013;339:823-826.
    • (2013) Science , vol.339 , pp. 823-826
    • Mali, P.1    Yang, L.2    Esvelt, K.M.3
  • 99
    • 0023600057 scopus 로고
    • Nucleotide sequence of the iap gene, responsible for alkaline phosphatase isozyme conversion in Escherichia coli, and identification of the gene product
    • Ishino Y, Shinagawa H, Makino K, et al. Nucleotide sequence of the iap gene, responsible for alkaline phosphatase isozyme conversion in Escherichia coli, and identification of the gene product. J Bacteriol. 1987;169:5429-5433.
    • (1987) J Bacteriol , vol.169 , pp. 5429-5433
    • Ishino, Y.1    Shinagawa, H.2    Makino, K.3
  • 100
    • 85020445347 scopus 로고    scopus 로고
    • A decade of discovery: CRISPR functions and applications
    • Barrangou R, Horvath P. A decade of discovery: CRISPR functions and applications. Nat Microbiol. 2017;2. 17092
    • (2017) Nat Microbiol , vol.2 , pp. 17092
    • Barrangou, R.1    Horvath, P.2
  • 101
    • 84902096048 scopus 로고    scopus 로고
    • Development and applications of CRISPR-Cas9 for genome engineering
    • Hsu PD, Lander ES, Zhang F. Development and applications of CRISPR-Cas9 for genome engineering. Cell. 2014;157:1262-1278.
    • (2014) Cell , vol.157 , pp. 1262-1278
    • Hsu, P.D.1    Lander, E.S.2    Zhang, F.3
  • 102
    • 85048050617 scopus 로고    scopus 로고
    • Nature, News 6 October, [3 April 2018]
    • Reardon S. Gene-editing record smashed in pigs. Nature. News 6 October 2015; http://www.nature.com/news/gene-editing-record-smashed-in-pigs-1.18525 [3 April 2018].
    • (2015) Gene-editing record smashed in pigs
    • Reardon, S.1
  • 103
    • 85027324183 scopus 로고    scopus 로고
    • Inactivation of porcine endogenous retrovirus in pigs using CRISPR-Cas9
    • Niu D, Wei H-J, Lin L, et al. Inactivation of porcine endogenous retrovirus in pigs using CRISPR-Cas9. Science. 2017;357:1303-1307.
    • (2017) Science , vol.357 , pp. 1303-1307
    • Niu, D.1    Wei, H.-J.2    Lin, L.3
  • 104
    • 85007513300 scopus 로고    scopus 로고
    • CRISPR gene-editing tested in a person for the first time
    • Cyranoski D. CRISPR gene-editing tested in a person for the first time. Nature. 2016;539:479.
    • (2016) Nature , vol.539 , pp. 479
    • Cyranoski, D.1
  • 105
    • 85048041068 scopus 로고    scopus 로고
    • [5 December
    • US National Library of Medicine, Clinical Trials Website. http://www.clinicaltrials.gov [5 December 2017].
    • (2017) Clinical Trials Website
  • 106
    • 85029315887 scopus 로고    scopus 로고
    • Correction of a splicing defect in a mouse model of congenital muscular dystrophy type 1A using a homology-directed-repair-independent mechanism
    • Kemaladewi DU, Maino E, Hyatt E, et al. Correction of a splicing defect in a mouse model of congenital muscular dystrophy type 1A using a homology-directed-repair-independent mechanism. Nat Med. 2017;23:984-989.
    • (2017) Nat Med , vol.23 , pp. 984-989
    • Kemaladewi, D.U.1    Maino, E.2    Hyatt, E.3
  • 107
    • 84960389900 scopus 로고    scopus 로고
    • Engineered viruses as genome editing devices
    • Chen X, Goncalves MA. Engineered viruses as genome editing devices. Mol Ther. 2016;24:447-454.
    • (2016) Mol Ther , vol.24 , pp. 447-454
    • Chen, X.1    Goncalves, M.A.2
  • 108
    • 84902095353 scopus 로고    scopus 로고
    • Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype
    • Yin H, Xue W, Chen S, et al. Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype. Nat Biotechnol. 2014;32:551-553.
    • (2014) Nat Biotechnol , vol.32 , pp. 551-553
    • Yin, H.1    Xue, W.2    Chen, S.3
  • 109
    • 84930618439 scopus 로고    scopus 로고
    • CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes
    • Liang P, Xu Y, Zhang X, et al. CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes. Protein Cell. 2015;6:363-372.
    • (2015) Protein Cell , vol.6 , pp. 363-372
    • Liang, P.1    Xu, Y.2    Zhang, X.3
  • 110
    • 85048032828 scopus 로고    scopus 로고
    • Science News, [Day Month Year]
    • Servick K. First US team to gene-edit human embryos revealed. Science News doi: 10.1126/science.aan7170. http://www.sciencemag.org/news/2017/07/first-us-team-gene-edit-human-embryos-revealed [Day Month Year].
    • First US team to gene-edit human embryos revealed
    • Servick, K.1
  • 111
    • 85026999487 scopus 로고    scopus 로고
    • Correction of a pathogenic gene mutation in human embryos
    • Ma H, Marti-Gutierrez N, Park SW, et al. Correction of a pathogenic gene mutation in human embryos. Nature. 2017;548:413-419.
    • (2017) Nature , vol.548 , pp. 413-419
    • Ma, H.1    Marti-Gutierrez, N.2    Park, S.W.3
  • 112
    • 84928775204 scopus 로고    scopus 로고
    • Don't edit the human germ line
    • Lanphier E, Urnov F, Haecker SE, et al. Don't edit the human germ line. Nature. 2015;519:410-411.
    • (2015) Nature , vol.519 , pp. 410-411
    • Lanphier, E.1    Urnov, F.2    Haecker, S.E.3
  • 113
    • 84928775846 scopus 로고    scopus 로고
    • Biotechnology. A prudent path forward for genomic engineering and germline gene modification
    • Baltimore D, Berg P, Botchan M, et al. Biotechnology. A prudent path forward for genomic engineering and germline gene modification. Science. 2015;348:36-38.
    • (2015) Science , vol.348 , pp. 36-38
    • Baltimore, D.1    Berg, P.2    Botchan, M.3
  • 114
    • 85047006969 scopus 로고    scopus 로고
    • A CRISPR new world: attitudes in the public toward innovations in human genetic modification
    • Weisberg SM, Badgio D, Chatterjee A. A CRISPR new world: attitudes in the public toward innovations in human genetic modification. Front Public Health. 2017;5:117.
    • (2017) Front Public Health , vol.5 , pp. 117
    • Weisberg, S.M.1    Badgio, D.2    Chatterjee, A.3
  • 117
    • 84904544984 scopus 로고    scopus 로고
    • Non-viral vectors for gene-based therapy
    • Yin H, Kanasty RL, Eltoukhy AA, et al. Non-viral vectors for gene-based therapy. Nat Rev Genet. 2014;15:541-555.
    • (2014) Nat Rev Genet , vol.15 , pp. 541-555
    • Yin, H.1    Kanasty, R.L.2    Eltoukhy, A.A.3
  • 118
    • 76349111051 scopus 로고    scopus 로고
    • Rational design of cationic lipids for siRNA delivery
    • Semple SC, Akinc A, Chen J, et al. Rational design of cationic lipids for siRNA delivery. Nat Biotechnol. 2010;28:172-176.
    • (2010) Nat Biotechnol , vol.28 , pp. 172-176
    • Semple, S.C.1    Akinc, A.2    Chen, J.3
  • 119
    • 85017559707 scopus 로고    scopus 로고
    • Lipid nanoparticle systems for enabling gene therapies
    • Cullis PR, Hope MJ. Lipid nanoparticle systems for enabling gene therapies. Mol Ther. 2017;25:1467-1475.
    • (2017) Mol Ther , vol.25 , pp. 1467-1475
    • Cullis, P.R.1    Hope, M.J.2
  • 120
    • 84942046481 scopus 로고    scopus 로고
    • Nucleic acid therapeutics using polyplexes: a journey of 50 years (and beyond)
    • Lachelt U, Wagner E. Nucleic acid therapeutics using polyplexes: a journey of 50 years (and beyond). Chem Rev. 2015;115:11043-11078.
    • (2015) Chem Rev , vol.115 , pp. 11043-11078
    • Lachelt, U.1    Wagner, E.2
  • 121
    • 0035135747 scopus 로고    scopus 로고
    • Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics
    • Kay MA, Glorioso JC, Naldini L. Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics. Nat Med. 2001;7:33-40.
    • (2001) Nat Med , vol.7 , pp. 33-40
    • Kay, M.A.1    Glorioso, J.C.2    Naldini, L.3
  • 122
    • 22244459917 scopus 로고    scopus 로고
    • Gene therapy: twenty-first century medicine
    • Verma IM, Weitzman MD. Gene therapy: twenty-first century medicine. Annu Rev Biochem. 2005;74:711-738.
    • (2005) Annu Rev Biochem , vol.74 , pp. 711-738
    • Verma, I.M.1    Weitzman, M.D.2
  • 123
    • 77958536106 scopus 로고    scopus 로고
    • Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA
    • Warren L, Manos PD, Ahfeldt T, et al. Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA. Cell Stem Cell. 2010;7:618-630.
    • (2010) Cell Stem Cell , vol.7 , pp. 618-630
    • Warren, L.1    Manos, P.D.2    Ahfeldt, T.3
  • 124
    • 72849137462 scopus 로고    scopus 로고
    • Adenoviral gene delivery can reprogram human fibroblasts to induced pluripotent stem cells
    • Zhou W, Freed CR. Adenoviral gene delivery can reprogram human fibroblasts to induced pluripotent stem cells. Stem Cells. 2009;27:2667-2674.
    • (2009) Stem Cells , vol.27 , pp. 2667-2674
    • Zhou, W.1    Freed, C.R.2
  • 125
    • 66049143859 scopus 로고    scopus 로고
    • Generation of human induced pluripotent stem cells by direct delivery of reprogramming proteins
    • Kim D, Kim C-H, Moon J-I, et al. Generation of human induced pluripotent stem cells by direct delivery of reprogramming proteins. Cell Stem Cell. 2009;4:472-476.
    • (2009) Cell Stem Cell , vol.4 , pp. 472-476
    • Kim, D.1    Kim, C.-H.2    Moon, J.-I.3
  • 126
    • 65649116572 scopus 로고    scopus 로고
    • Human induced pluripotent stem cells free of vector and transgene sequences
    • Yu J, Hu K, Smuga-Otto K, et al. Human induced pluripotent stem cells free of vector and transgene sequences. Science. 2009;324:797-801.
    • (2009) Science , vol.324 , pp. 797-801
    • Yu, J.1    Hu, K.2    Smuga-Otto, K.3
  • 127
    • 84993978039 scopus 로고    scopus 로고
    • Sendai virus, an RNA virus with no risk of genomic integration, delivers CRISPR/Cas9 for efficient gene editing
    • Park A, Hong P, Won ST, et al. Sendai virus, an RNA virus with no risk of genomic integration, delivers CRISPR/Cas9 for efficient gene editing. Mol Ther Methods Clin Dev. 2016;3. 16057
    • (2016) Mol Ther Methods Clin Dev , vol.3 , pp. 16057
    • Park, A.1    Hong, P.2    Won, S.T.3
  • 128
    • 84962593365 scopus 로고    scopus 로고
    • Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles
    • Wang M, Zuris JA, Meng F, et al. Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles. Proc Natl Acad Sci U S A. 2016;113:2868-2873.
    • (2016) Proc Natl Acad Sci U S A , vol.113 , pp. 2868-2873
    • Wang, M.1    Zuris, J.A.2    Meng, F.3
  • 129
    • 85018898150 scopus 로고    scopus 로고
    • Efficient genome editing in the mouse brain by local delivery of engineered Cas9 ribonucleoprotein complexes
    • Staahl BT, Benekareddy M, Coulon-Bainier C, et al. Efficient genome editing in the mouse brain by local delivery of engineered Cas9 ribonucleoprotein complexes. Nat Biotechnol. 2017;35:431-434.
    • (2017) Nat Biotechnol , vol.35 , pp. 431-434
    • Staahl, B.T.1    Benekareddy, M.2    Coulon-Bainier, C.3
  • 130
    • 85020721608 scopus 로고    scopus 로고
    • Integrase-deficient lentiviral vector as an all-in-one platform for highly efficient CRISPR/Cas9-mediated gene editing
    • Ortinski PI, O'Donovan B, Dong X, et al. Integrase-deficient lentiviral vector as an all-in-one platform for highly efficient CRISPR/Cas9-mediated gene editing. Mol Ther Methods Clin Dev. 2017;5:153-164.
    • (2017) Mol Ther Methods Clin Dev , vol.5 , pp. 153-164
    • Ortinski, P.I.1    O'Donovan, B.2    Dong, X.3
  • 131
    • 85019953441 scopus 로고    scopus 로고
    • Hit and go CAS9 delivered through a lentiviral based self-limiting circuit
    • Petris G, Casini A, Montagna C, et al. Hit and go CAS9 delivered through a lentiviral based self-limiting circuit. Nat Commun. 2017;8. 15334
    • (2017) Nat Commun , vol.8 , pp. 15334
    • Petris, G.1    Casini, A.2    Montagna, C.3
  • 132
    • 85048041053 scopus 로고    scopus 로고
    • [5 December
    • National Institutes of Health Research, UK Clinical Trials Gateway. http://www.ukctg.nihr.ac.uk [5 December 2017].
    • (2017) UK Clinical Trials Gateway
  • 134
    • 85048047072 scopus 로고    scopus 로고
    • [5 December
    • University Medical Centre Freiburg and the German Cochrane Center, German Clinical Trials Register. https://www.drks.de/drks_web [5 December 2017].
    • (2017) German Clinical Trials Register
  • 135
    • 85048048494 scopus 로고    scopus 로고
    • [5 December
    • Swiss Expert Committee for Biosafety, Gene Therapy. http://www.efbs.admin.ch/en/topics/gene-therapy.html [5 December 2017].
    • (2017) Gene Therapy
  • 136
    • 85046707969 scopus 로고    scopus 로고
    • [5 December
    • European Medicines Agency, EU Clinical Trials Register. https://www.clinicaltrialsregister.eu [5 December 2017].
    • (2017) EU Clinical Trials Register
  • 139
    • 85048037623 scopus 로고    scopus 로고
    • [5 December
    • The Chinese Clinical Test Registration Center, Chinese Clinical Trial Registry. http://www.chictr.org.cn/enIndex.aspx [5 December 2017].
    • (2017) Chinese Clinical Trial Registry
  • 140
    • 85048038927 scopus 로고    scopus 로고
    • [5 December
    • National Institutes of Public Health Japan, NIPH Clinical Trials Search. http://rctportal.niph.go.jp/en [5 December 2017].
    • (2017) NIPH Clinical Trials Search
  • 142
    • 85040735420 scopus 로고    scopus 로고
    • Clinical and immunologic biomarkers for histologic regression of high-grade cervical dysplasia and clearance of HPV16 and HPV18 after immunotherapy
    • Morrow MP, Kraynyak KA, Sylvester AJ, et al. Clinical and immunologic biomarkers for histologic regression of high-grade cervical dysplasia and clearance of HPV16 and HPV18 after immunotherapy. Clin Cancer Res. 2018;24:276-294.
    • (2018) Clin Cancer Res , vol.24 , pp. 276-294
    • Morrow, M.P.1    Kraynyak, K.A.2    Sylvester, A.J.3
  • 143
    • 84947869406 scopus 로고    scopus 로고
    • Safety, efficacy, and immunogenicity of VGX-3100, a therapeutic synthetic DNA vaccine targeting human papillomavirus 16 and 18 E6 and E7 proteins for cervical intraepithelial neoplasia 2/3: a randomised, double-blind, placebo-controlled phase 2b trial
    • Trimble CL, Morrow MP, Kraynyak KA, et al. Safety, efficacy, and immunogenicity of VGX-3100, a therapeutic synthetic DNA vaccine targeting human papillomavirus 16 and 18 E6 and E7 proteins for cervical intraepithelial neoplasia 2/3: a randomised, double-blind, placebo-controlled phase 2b trial. Lancet. 2015;386:2078-2088.
    • (2015) Lancet , vol.386 , pp. 2078-2088
    • Trimble, C.L.1    Morrow, M.P.2    Kraynyak, K.A.3
  • 145
    • 85023198537 scopus 로고    scopus 로고
    • Employing RNA viruses to fight cancer: novel insights into oncolytic virotherapy
    • Masemann D, Boergeling Y, Ludwig S. Employing RNA viruses to fight cancer: novel insights into oncolytic virotherapy. Biol Chem. 2017;398:891-909.
    • (2017) Biol Chem , vol.398 , pp. 891-909
    • Masemann, D.1    Boergeling, Y.2    Ludwig, S.3
  • 146
    • 84988329719 scopus 로고    scopus 로고
    • Advanced new strategies for metastatic cancer treatment by therapeutic stem cells and oncolytic virotherapy
    • Park GT, Choi KC. Advanced new strategies for metastatic cancer treatment by therapeutic stem cells and oncolytic virotherapy. Oncotarget. 2016;7:58684-58695.
    • (2016) Oncotarget , vol.7 , pp. 58684-58695
    • Park, G.T.1    Choi, K.C.2
  • 147
    • 85017113587 scopus 로고    scopus 로고
    • Oncolytic virotherapy: a contest between apples and oranges
    • Russell SJ, Peng KW. Oncolytic virotherapy: a contest between apples and oranges. Mol Ther. 2017;25:1107-1116.
    • (2017) Mol Ther , vol.25 , pp. 1107-1116
    • Russell, S.J.1    Peng, K.W.2
  • 148
    • 84977656495 scopus 로고    scopus 로고
    • AMGEN website Press Release 27 October, [30 January, 2018]
    • Amgen. FDA Approves IMLYGIC™ (Talimogene Laherparepvec) As First Oncolytic Viral Therapy In The US. AMGEN website: Press Release 27 October 2015. http://www.amgen.com/media/news-releases/2015/10/fda-approves-imlygic-talimogene-laherparepvec-as-first-oncolytic-viral-therapy-in-the-us/ [30 January, 2018].
    • (2015) FDA Approves IMLYGIC™ (Talimogene Laherparepvec) As First Oncolytic Viral Therapy In The US
  • 149
    • 85017348801 scopus 로고    scopus 로고
    • Cardiovascular gene therapy: past, present, and future
    • Yla-Herttuala S, Baker AH. Cardiovascular gene therapy: past, present, and future. Mol Ther. 2017;25:1095-1106.
    • (2017) Mol Ther , vol.25 , pp. 1095-1106
    • Yla-Herttuala, S.1    Baker, A.H.2
  • 150
    • 0037101969 scopus 로고    scopus 로고
    • Enhanced delivery of naked DNA to the skin by non-invasive in vivo electroporation
    • Zhang L, Nolan E, Kreitschitz S, et al. Enhanced delivery of naked DNA to the skin by non-invasive in vivo electroporation. Biochim Biophys Acta. 2002;1572:1-9.
    • (2002) Biochim Biophys Acta , vol.1572 , pp. 1-9
    • Zhang, L.1    Nolan, E.2    Kreitschitz, S.3
  • 151
    • 33747195353 scopus 로고    scopus 로고
    • Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors
    • Takahashi K, Yamanaka S. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell. 2006;126:663-676.
    • (2006) Cell , vol.126 , pp. 663-676
    • Takahashi, K.1    Yamanaka, S.2
  • 152
    • 84923014395 scopus 로고    scopus 로고
    • Human embryonic stem cell-derived retinal pigment epithelium in patients with age-related macular degeneration and Stargardt's macular dystrophy: follow-up of two open-label phase 1/2 studies
    • Schwartz SD, Regillo CD, Lam BL, et al. Human embryonic stem cell-derived retinal pigment epithelium in patients with age-related macular degeneration and Stargardt's macular dystrophy: follow-up of two open-label phase 1/2 studies. Lancet. 2015;385:509-516.
    • (2015) Lancet , vol.385 , pp. 509-516
    • Schwartz, S.D.1    Regillo, C.D.2    Lam, B.L.3
  • 153
    • 85025475102 scopus 로고    scopus 로고
    • Stem cell therapies for reversing vision loss
    • Higuchi A, Kumar SS, Benelli G, et al. Stem cell therapies for reversing vision loss. Trends Biotechnol. 2017;35:1102-1117.
    • (2017) Trends Biotechnol , vol.35 , pp. 1102-1117
    • Higuchi, A.1    Kumar, S.S.2    Benelli, G.3
  • 154
    • 84955656032 scopus 로고    scopus 로고
    • Stem cells in clinical trials for treatment of retinal degeneration
    • Klassen H. Stem cells in clinical trials for treatment of retinal degeneration. Expert Opin Biol Ther. 2016;16:7-14.
    • (2016) Expert Opin Biol Ther , vol.16 , pp. 7-14
    • Klassen, H.1
  • 155
    • 85016968122 scopus 로고    scopus 로고
    • Systemic injection of RPE65-programmed bone marrow-derived cells prevents progression of chronic retinal degeneration
    • Qi X, Pay SL, Yan Y, et al. Systemic injection of RPE65-programmed bone marrow-derived cells prevents progression of chronic retinal degeneration. Mol Ther. 2017;25:917-927.
    • (2017) Mol Ther , vol.25 , pp. 917-927
    • Qi, X.1    Pay, S.L.2    Yan, Y.3
  • 156
    • 84907752479 scopus 로고    scopus 로고
    • Japan stem-cell trial stirs envy
    • Reardon S, Cyranoski D. Japan stem-cell trial stirs envy. Nature. 2014;513:287-288.
    • (2014) Nature , vol.513 , pp. 287-288
    • Reardon, S.1    Cyranoski, D.2
  • 157
    • 84965092064 scopus 로고    scopus 로고
    • RIKEN suspends first clinical trial involving induced pluripotent stem cells
    • Garber K. RIKEN suspends first clinical trial involving induced pluripotent stem cells. Nat Biotechnol. 2015;33:890-891.
    • (2015) Nat Biotechnol , vol.33 , pp. 890-891
    • Garber, K.1
  • 158
    • 85015803917 scopus 로고    scopus 로고
    • Autologous induced stem-cell-derived retinal cells for macular degeneration
    • Mandai M, Watanabe A, Kurimoto Y, et al. Autologous induced stem-cell-derived retinal cells for macular degeneration. N Engl J Med. 2017;376:1038-1046.
    • (2017) N Engl J Med , vol.376 , pp. 1038-1046
    • Mandai, M.1    Watanabe, A.2    Kurimoto, Y.3
  • 159
    • 85015155107 scopus 로고    scopus 로고
    • iPS cell therapy reported safe
    • Normile D. iPS cell therapy reported safe. Science. 2017;355:1109-1110.
    • (2017) Science , vol.355 , pp. 1109-1110
    • Normile, D.1
  • 160
    • 85006285022 scopus 로고    scopus 로고
    • Induced pluripotent stem cell technology: a decade of progress
    • Shi Y, Inoue H, Wu JC, et al. Induced pluripotent stem cell technology: a decade of progress. Nat Rev Drug Discov. 2017;16:115-130.
    • (2017) Nat Rev Drug Discov , vol.16 , pp. 115-130
    • Shi, Y.1    Inoue, H.2    Wu, J.C.3
  • 161
    • 0034328925 scopus 로고    scopus 로고
    • Gene therapy: trials and tribulations
    • Somia N, Verma IM. Gene therapy: trials and tribulations. Nat Rev Genet. 2000;1:91-99.
    • (2000) Nat Rev Genet , vol.1 , pp. 91-99
    • Somia, N.1    Verma, I.M.2
  • 162
    • 70349918248 scopus 로고    scopus 로고
    • Evading the immune response upon in vivo gene therapy with viral vectors
    • Sack BK, Herzog RW. Evading the immune response upon in vivo gene therapy with viral vectors. Curr Opin Mol Ther. 2009;11:493-503.
    • (2009) Curr Opin Mol Ther , vol.11 , pp. 493-503
    • Sack, B.K.1    Herzog, R.W.2
  • 163
    • 84877930020 scopus 로고    scopus 로고
    • Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells
    • Martino AT, Basner-Tschakarjan E, Markusic DM, et al. Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells. Blood. 2013;121:2224-2233.
    • (2013) Blood , vol.121 , pp. 2224-2233
    • Martino, A.T.1    Basner-Tschakarjan, E.2    Markusic, D.M.3
  • 164
    • 84986290412 scopus 로고    scopus 로고
    • Gene therapy: industrial strength
    • Bender E. Gene therapy: industrial strength. Nature. 2016;537:S57-S59.
    • (2016) Nature , vol.537 , pp. S57-S59
    • Bender, E.1
  • 165
    • 84973360045 scopus 로고    scopus 로고
    • MEDICINE. Paying for future success in gene therapy
    • Orkin SH, Reilly P. MEDICINE. Paying for future success in gene therapy. Science. 2016;352:1059-1061.
    • (2016) Science , vol.352 , pp. 1059-1061
    • Orkin, S.H.1    Reilly, P.2
  • 166
    • 85020934874 scopus 로고    scopus 로고
    • Supply of promising T cell therapy is strained
    • Couzin-Frankel J. Supply of promising T cell therapy is strained. Science. 2017;356:1112-1113.
    • (2017) Science , vol.356 , pp. 1112-1113
    • Couzin-Frankel, J.1
  • 167
    • 33847432207 scopus 로고
    • Will society be prepared?
    • Nirenberg MW. Will society be prepared? Science. 1967;157:633.
    • (1967) Science , vol.157 , pp. 633
    • Nirenberg, M.W.1


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.