{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2022,4,4]],"date-time":"2022-04-04T03:22:54Z","timestamp":1649042574965},"reference-count":24,"publisher":"Japan Society for Clinical Immunology","issue":"3","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Jpn. J. Clin. Immunol.","Jpn.J.clin.Immun"],"published-print":{"date-parts":[[2013]]},"DOI":"10.2177\/jsci.36.148","type":"journal-article","created":{"date-parts":[[2013,6,29]],"date-time":"2013-06-29T22:45:54Z","timestamp":1372545954000},"page":"148-155","source":"Crossref","is-referenced-by-count":0,"title":["Current status and future prospects of stem cell gene therapy for primary immunodeficiency"],"prefix":"10.2177","volume":"36","author":[{"given":"Toru","family":"UCHIYAMA","sequence":"first","affiliation":[{"name":"Department of Human genetics, National Center for Child Health and Development"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Masafumi","family":"ONODERA","sequence":"additional","affiliation":[{"name":"Department of Human genetics, National Center for Child Health and Development"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"972","reference":[{"key":"1","unstructured":"1) Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M. : Gene therapy for primary immunodeficiencies. <i>Immunol Allergy Clin North Am<\/i>. <b>30<\/b>(2) : 237-248, 2010."},{"key":"2","unstructured":"2) Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, et al. : Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science. <b>288<\/b>(5466) : 669-672, 2000."},{"key":"3","unstructured":"3) Gaspar HB, Parsley KL, Howe S, et al. : Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. <i>Lancet<\/i>. <b>364<\/b>(9452) : 2181-2187, 2004."},{"key":"4","unstructured":"4) Cavazzana-Calvo M, Fischer A, Hacein-Bey-Abina S, Aiuti A. : Gene therapy for primary immunodeficiencies: Part 1. <i>Curr Opin Immunol<\/i>. <b>24<\/b>(5) : 580-584, 2012."},{"key":"5","unstructured":"5) Hacein-Bey-Abina S, Hauer J, Lim A, et al. : Efficacy of gene therapy for X-linked severe combined immunodeficiency. <i>N Engl J Med<\/i>. <b>363<\/b>(4) : 355-364, 2010."},{"key":"6","unstructured":"6) Hacein-Bey-Abina S, Garrigue A, Wang GP, et al. : Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. <i>J Clin Invest<\/i>. <b>118<\/b>(9) : 3132-3142, 2008."},{"key":"7","unstructured":"7) Howe SJ, Mansour MR, Schwarzwaelder K, et al. : Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. <i>J Clin Invest<\/i>. <b>118<\/b>(9) : 3143-3150, 2008."},{"key":"8","unstructured":"8) Thornhill SI, Schambach A, Howe SJ, et al. : Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency. <i>Mol Ther.<\/i> <b>16<\/b>(3) : 590-598, 2008."},{"key":"9","unstructured":"9) Blaese RM, Culver KW, Miller AD, et al. : T lymphocyte-directed gene therapy for ADA-SCID : initial trial results after 4 years. <i>Science<\/i>. <b>270<\/b>(5235) : 475-480, 1995."},{"key":"10","unstructured":"10) Aiuti A, Cattaneo F, Galimberti S, et al. : Gene therapy for immunodeficiency due to adenosine deaminase deficiency. <i>N Engl J Med<\/i>. <b>360<\/b>(5) : 447-458, 2009."},{"key":"11","unstructured":"11) Gaspar HB, Aiuti A, Porta F, Candotti F, Hershfield MS, Notarangelo LD. : How I treat ADA deficiency. <i>Blood<\/i>. <b>114<\/b>(17) : 3524-3532, 2009."},{"key":"12","unstructured":"12) Konno A, Wada T, Schurman SH, et al. : Differential contribution of Wiskott-Aldrich syndrome protein to selective advantage in T- and B-cell lineages. <i>Blood<\/i>. <b>103<\/b>(2) : 676-678, 2004."},{"key":"13","unstructured":"13) Boztug K, Schmidt M, Schwarzer A, et al. : Stem-cell gene therapy for the Wiskott-Aldrich syndrome. <i>N Engl J Med<\/i>. <b>363<\/b>(20) : 1918-1927, 2010."},{"key":"14","unstructured":"14) Marangoni F, Bosticardo M, Charrier S, et al. : Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models. <i>Mol Ther<\/i>. <b>17<\/b>(6) : 1073-1082, 2009."},{"key":"15","unstructured":"15) Scaramuzza S, Biasco L, Ripamonti A, et al. : Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome. <i>Mol Ther<\/i>. <b>21<\/b>(1) : 175-184, 2013."},{"key":"16","unstructured":"16) Aiuti A, Bacchetta R, Seger R, Villa A, Cavazzana-Calvo M. : Gene therapy for primary immunodeficiencies : Part 2. <i>Curr Opin Immunol<\/i>. <b>24<\/b>(5) : 585-591, 2012."},{"key":"17","unstructured":"17) Kang EM, Choi U, Theobald N, et al. : Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils. <i>Blood<\/i>. <b>115<\/b>(4) : 783-791, 2010."},{"key":"18","unstructured":"18) Kang HJ, Bartholomae CC, Paruzynski A, et al. : Retroviral gene therapy for X-linked chronic granulomatous disease : results from phase I\/II trial. <i>Mol Ther<\/i>. <b>19<\/b>(11) : 2092-2101, 2011."},{"key":"19","unstructured":"19) 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. <i>Nat Med<\/i>. <b>12<\/b>(4) : 401-409, 2006."},{"key":"20","unstructured":"20) Cartier N, Hacein-Bey-Abina S, Bartholomae CC, et al. : Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. <i>Science<\/i>. <b>326<\/b>(5954) : 818-823, 2009."},{"key":"21","unstructured":"21) Nathwani AC, Tuddenham EG, Rangarajan S, et al. : Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. <i>N Engl J Med<\/i>. <b>365<\/b>(25) : 2357-2365, 2011."},{"key":"22","unstructured":"22) Moiani A, Paleari Y, Sartori D, et al. : Lentiviral vector integration in the human genome induces alternative splicing and generates aberrant transcripts. <i>J Clin Invest<\/i>. <b>122<\/b>(5) : 1653-1666, 2012."},{"key":"23","unstructured":"23) Cesana D, Sgualdino J, Rudilosso L, Merella S, Naldini L, Montini E. : Whole transcriptome characterization of aberrant splicing events induced by lentiviral vector integrations. <i>J Clin Invest<\/i>. <b>122<\/b>(5) : 1667-1676, 2012."},{"key":"24","unstructured":"24) Cavazzana-Calvo M, Payen E, Negre O, et al. : Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia. <i>Nature<\/i>. <b>467<\/b>(7313) : 318-322, 2010."}],"container-title":["Japanese Journal of Clinical Immunology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.jstage.jst.go.jp\/article\/jsci\/36\/3\/36_148\/_pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2021,5,20]],"date-time":"2021-05-20T04:51:45Z","timestamp":1621486305000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.jstage.jst.go.jp\/article\/jsci\/36\/3\/36_148\/_article\/-char\/ja\/"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2013]]},"references-count":24,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2013]]}},"URL":"https:\/\/doi.org\/10.2177\/jsci.36.148","relation":{},"ISSN":["0911-4300","1349-7413"],"issn-type":[{"value":"0911-4300","type":"print"},{"value":"1349-7413","type":"electronic"}],"subject":[],"published":{"date-parts":[[2013]]}}}