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Items: 1 to 20 of 115

1.

Efficacy of gene therapy for X-linked severe combined immunodeficiency.

Hacein-Bey-Abina S, Hauer J, Lim A, Picard C, Wang GP, Berry CC, Martinache C, Rieux-Laucat F, Latour S, Belohradsky BH, Leiva L, Sorensen R, Debré M, Casanova JL, Blanche S, Durandy A, Bushman FD, Fischer A, Cavazzana-Calvo M.

N Engl J Med. 2010 Jul 22;363(4):355-64. doi: 10.1056/NEJMoa1000164.

2.

Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy.

Hacein-Bey-Abina S, Le Deist F, Carlier F, Bouneaud C, Hue C, De Villartay JP, Thrasher AJ, Wulffraat N, Sorensen R, Dupuis-Girod S, Fischer A, Davies EG, Kuis W, Leiva L, Cavazzana-Calvo M.

N Engl J Med. 2002 Apr 18;346(16):1185-93.

3.

Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector.

Gaspar HB, Parsley KL, Howe S, King D, Gilmour KC, Sinclair J, Brouns G, Schmidt M, Von Kalle C, Barington T, Jakobsen MA, Christensen HO, Al Ghonaium A, White HN, Smith JL, Levinsky RJ, Ali RR, Kinnon C, Thrasher AJ.

Lancet. 2004 Dec 18-31;364(9452):2181-7.

PMID:
15610804
4.

Treatment of an infant with X-linked severe combined immunodeficiency (SCID-X1) by gene therapy in Australia.

Ginn SL, Curtin JA, Kramer B, Smyth CM, Wong M, Kakakios A, McCowage GB, Watson D, Alexander SI, Latham M, Cunningham SC, Zheng M, Hobson L, Rowe PB, Fischer A, Cavazzana-Calvo M, Hacein-Bey-Abina S, Alexander IE.

Med J Aust. 2005 May 2;182(9):458-63.

PMID:
15865589
5.

Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease.

Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, Gross F, Yvon E, Nusbaum P, Selz F, Hue C, Certain S, Casanova JL, Bousso P, Deist FL, Fischer A.

Science. 2000 Apr 28;288(5466):669-72.

6.

A modified γ-retrovirus vector for X-linked severe combined immunodeficiency.

Hacein-Bey-Abina S, Pai SY, Gaspar HB, Armant M, Berry CC, Blanche S, Bleesing J, Blondeau J, de Boer H, Buckland KF, Caccavelli L, Cros G, De Oliveira S, Fernández KS, Guo D, Harris CE, Hopkins G, Lehmann LE, Lim A, London WB, van der Loo JC, Malani N, Male F, Malik P, Marinovic MA, McNicol AM, Moshous D, Neven B, Oleastro M, Picard C, Ritz J, Rivat C, Schambach A, Shaw KL, Sherman EA, Silberstein LE, Six E, Touzot F, Tsytsykova A, Xu-Bayford J, Baum C, Bushman FD, Fischer A, Kohn DB, Filipovich AH, Notarangelo LD, Cavazzana M, Williams DA, Thrasher AJ.

N Engl J Med. 2014 Oct 9;371(15):1407-17. doi: 10.1056/NEJMoa1404588.

7.

Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency.

Gaspar HB, Cooray S, Gilmour KC, Parsley KL, Adams S, Howe SJ, Al Ghonaium A, Bayford J, Brown L, Davies EG, Kinnon C, Thrasher AJ.

Sci Transl Med. 2011 Aug 24;3(97):97ra79. doi: 10.1126/scitranslmed.3002715.

8.

Long-term immune reconstitution after anti-CD52-treated or anti-CD34-treated hematopoietic stem cell transplantation for severe T-lymphocyte immunodeficiency.

Slatter MA, Brigham K, Dickinson AM, Harvey HL, Barge D, Jackson A, Bown N, Flood TJ, Cant AJ, Abinun M, Gennery AR.

J Allergy Clin Immunol. 2008 Feb;121(2):361-7. Epub 2007 Dec 20.

PMID:
18086494
9.

Transplantation of X-linked severe combined immunodeficient dogs with CD34+ bone marrow cells.

Hartnett BJ, Yao D, Suter SE, Ellinwood NM, Henthorn PS, Moore PE, McSweeney PA, Nash RA, Brown JD, Weinberg KI, Felsburg PJ.

Biol Blood Marrow Transplant. 2002;8(4):188-97.

10.

Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

Aiuti A, Cattaneo F, Galimberti S, Benninghoff U, Cassani B, Callegaro L, Scaramuzza S, Andolfi G, Mirolo M, Brigida I, Tabucchi A, Carlucci F, Eibl M, Aker M, Slavin S, Al-Mousa H, Al Ghonaium A, Ferster A, Duppenthaler A, Notarangelo L, Wintergerst U, Buckley RH, Bregni M, Marktel S, Valsecchi MG, Rossi P, Ciceri F, Miniero R, Bordignon C, Roncarolo MG.

N Engl J Med. 2009 Jan 29;360(5):447-58. doi: 10.1056/NEJMoa0805817.

11.

Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency.

Buckley RH, Schiff SE, Schiff RI, Markert L, Williams LW, Roberts JL, Myers LA, Ward FE.

N Engl J Med. 1999 Feb 18;340(7):508-16.

12.

A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells.

Zhou S, Mody D, DeRavin SS, Hauer J, Lu T, Ma Z, Hacein-Bey Abina S, Gray JT, Greene MR, Cavazzana-Calvo M, Malech HL, Sorrentino BP.

Blood. 2010 Aug 12;116(6):900-8. doi: 10.1182/blood-2009-10-250209. Epub 2010 May 10.

13.

Effect of ex vivo culture of CD34+ bone marrow cells on immune reconstitution of XSCID dogs following allogeneic bone marrow transplantation.

Kennedy DR, McLellan K, Moore PF, Henthorn PS, Felsburg PJ.

Biol Blood Marrow Transplant. 2009 Jun;15(6):662-70. doi: 10.1016/j.bbmt.2009.03.014.

14.

[Transplantation of genetically modified cells in the treatment of children with SCID: great hopes and recent disappointments].

Smogorzewska EM, Weinberg KI, Kohn DB.

Med Wieku Rozwoj. 2003 Jan-Mar;7(1):27-34. Review. Polish.

PMID:
13130167
15.

Retroviral transduction of IL2RG into CD34(+) cells from X-linked severe combined immunodeficiency patients permits human T- and B-cell development in sheep chimeras.

Tsai EJ, Malech HL, Kirby MR, Hsu AP, Seidel NE, Porada CD, Zanjani ED, Bodine DM, Puck JM.

Blood. 2002 Jul 1;100(1):72-9.

16.

Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning.

Huston MW, van Til NP, Visser TP, Arshad S, Brugman MH, Cattoglio C, Nowrouzi A, Li Y, Schambach A, Schmidt M, Baum C, von Kalle C, Mavilio F, Zhang F, Blundell MP, Thrasher AJ, Verstegen MM, Wagemaker G.

Mol Ther. 2011 Oct;19(10):1867-77. doi: 10.1038/mt.2011.127. Epub 2011 Jul 12.

17.

Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction.

Gaspar HB, Cooray S, Gilmour KC, Parsley KL, Zhang F, Adams S, Bjorkegren E, Bayford J, Brown L, Davies EG, Veys P, Fairbanks L, Bordon V, Petropoulou T, Kinnon C, Thrasher AJ.

Sci Transl Med. 2011 Aug 24;3(97):97ra80. doi: 10.1126/scitranslmed.3002716. Erratum in: Sci Transl Med. 2013 Jan 16;5(168):168er1. Petropolou, Theoni [corrected to Petropoulou, Theoni].

18.

Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency.

De Ravin SS, Wu X, Moir S, Anaya-O'Brien S, Kwatemaa N, Littel P, Theobald N, Choi U, Su L, Marquesen M, Hilligoss D, Lee J, Buckner CM, Zarember KA, O'Connor G, McVicar D, Kuhns D, Throm RE, Zhou S, Notarangelo LD, Hanson IC, Cowan MJ, Kang E, Hadigan C, Meagher M, Gray JT, Sorrentino BP, Malech HL, Kardava L.

Sci Transl Med. 2016 Apr 20;8(335):335ra57. doi: 10.1126/scitranslmed.aad8856. Erratum in: Sci Transl Med. 2016 Jun 1;8(341):341er5.

19.

[Gene therapy of X-linked severe combined immunologic deficiency (SCID-X1)].

Hacein-Bey S, Gross F, Nusbaum P, Yvon E, Fischer A, Cavazzana-Calvo M.

Pathol Biol (Paris). 2001 Feb;49(1):57-66. Review. French.

PMID:
11265225
20.

Pretransplant mobilization with granulocyte colony-stimulating factor improves B-cell reconstitution by lentiviral vector gene therapy in SCID-X1 mice.

Huston MW, Riegman AR, Yadak R, van Helsdingen Y, de Boer H, van Til NP, Wagemaker G.

Hum Gene Ther. 2014 Oct;25(10):905-14. doi: 10.1089/hum.2014.101.

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