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Items: 1 to 20 of 216

1.

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.

2.

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.

3.

An experimental system for the evaluation of retroviral vector design to diminish the risk for proto-oncogene activation.

Ryu BY, Evans-Galea MV, Gray JT, Bodine DM, Persons DA, Nienhuis AW.

Blood. 2008 Feb 15;111(4):1866-75. Epub 2007 Nov 8.

4.

Gene therapy model of X-linked severe combined immunodeficiency using a modified foamy virus vector.

Horino S, Uchiyama T, So T, Nagashima H, Sun SL, Sato M, Asao A, Haji Y, Sasahara Y, Candotti F, Tsuchiya S, Kure S, Sugamura K, Ishii N.

PLoS One. 2013 Aug 21;8(8):e71594. doi: 10.1371/journal.pone.0071594. eCollection 2013.

5.

Correction of murine Rag2 severe combined immunodeficiency by lentiviral gene therapy using a codon-optimized RAG2 therapeutic transgene.

van Til NP, de Boer H, Mashamba N, Wabik A, Huston M, Visser TP, Fontana E, Poliani PL, Cassani B, Zhang F, Thrasher AJ, Villa A, Wagemaker G.

Mol Ther. 2012 Oct;20(10):1968-80. doi: 10.1038/mt.2012.110. Epub 2012 Jun 12.

6.

Lymphomagenesis in SCID-X1 mice following lentivirus-mediated phenotype correction independent of insertional mutagenesis and gammac overexpression.

Ginn SL, Liao SH, Dane AP, Hu M, Hyman J, Finnie JW, Zheng M, Cavazzana-Calvo M, Alexander SI, Thrasher AJ, Alexander IE.

Mol Ther. 2010 May;18(5):965-76. doi: 10.1038/mt.2010.50. Epub 2010 Mar 30.

7.
8.

Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency.

Carbonaro DA, Zhang L, Jin X, Montiel-Equihua C, Geiger S, Carmo M, Cooper A, Fairbanks L, Kaufman ML, Sebire NJ, Hollis RP, Blundell MP, Senadheera S, Fu PY, Sahaghian A, Chan RY, Wang X, Cornetta K, Thrasher AJ, Kohn DB, Gaspar HB.

Mol Ther. 2014 Mar;22(3):607-22. doi: 10.1038/mt.2013.265. Epub 2013 Nov 20.

9.

Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency.

Thornhill SI, Schambach A, Howe SJ, Ulaganathan M, Grassman E, Williams D, Schiedlmeier B, Sebire NJ, Gaspar HB, Kinnon C, Baum C, Thrasher AJ.

Mol Ther. 2008 Mar;16(3):590-8. doi: 10.1038/sj.mt.6300393. Epub 2008 Jan 8.

PMID:
18180772
10.

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
11.

Recombination-activating gene 1 (Rag1)-deficient mice with severe combined immunodeficiency treated with lentiviral gene therapy demonstrate autoimmune Omenn-like syndrome.

van Til NP, Sarwari R, Visser TP, Hauer J, Lagresle-Peyrou C, van der Velden G, Malshetty V, Cortes P, Jollet A, Danos O, Cassani B, Zhang F, Thrasher AJ, Fontana E, Poliani PL, Cavazzana M, Verstegen MM, Villa A, Wagemaker G.

J Allergy Clin Immunol. 2014 Apr;133(4):1116-23. doi: 10.1016/j.jaci.2013.10.009. Epub 2013 Dec 9.

PMID:
24332219
12.

Comparison of five retrovirus vectors containing the human IL-2 receptor gamma chain gene for their ability to restore T and B lymphocytes in the X-linked severe combined immunodeficiency mouse model.

Avilés Mendoza GJ, Seidel NE, Otsu M, Anderson SM, Simon-Stoos K, Herrera A, Hoogstraten-Miller S, Malech HL, Candotti F, Puck JM, Bodine DM.

Mol Ther. 2001 Apr;3(4):565-73.

13.

Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer.

Pike-Overzet K, Rodijk M, Ng YY, Baert MR, Lagresle-Peyrou C, Schambach A, Zhang F, Hoeben RC, Hacein-Bey-Abina S, Lankester AC, Bredius RG, Driessen GJ, Thrasher AJ, Baum C, Cavazzana-Calvo M, van Dongen JJ, Staal FJ.

Leukemia. 2011 Sep;25(9):1471-83. doi: 10.1038/leu.2011.106. Epub 2011 May 27.

PMID:
21617701
14.

Ectopic retroviral expression of LMO2, but not IL2Rgamma, blocks human T-cell development from CD34+ cells: implications for leukemogenesis in gene therapy.

Pike-Overzet K, de Ridder D, Weerkamp F, Baert MR, Verstegen MM, Brugman MH, Howe SJ, Reinders MJ, Thrasher AJ, Wagemaker G, van Dongen JJ, Staal FJ.

Leukemia. 2007 Apr;21(4):754-63. Epub 2007 Feb 1.

PMID:
17268520
15.

Transcriptional targeting of lentiviral vectors by long terminal repeat enhancer replacement.

Lotti F, Menguzzato E, Rossi C, Naldini L, Ailles L, Mavilio F, Ferrari G.

J Virol. 2002 Apr;76(8):3996-4007.

16.

LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1.

Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, Lim A, Osborne CS, Pawliuk R, Morillon E, Sorensen R, Forster A, Fraser P, Cohen JI, de Saint Basile G, Alexander I, Wintergerst U, Frebourg T, Aurias A, Stoppa-Lyonnet D, Romana S, Radford-Weiss I, Gross F, Valensi F, Delabesse E, Macintyre E, Sigaux F, Soulier J, Leiva LE, Wissler M, Prinz C, Rabbitts TH, Le Deist F, Fischer A, Cavazzana-Calvo M.

Science. 2003 Oct 17;302(5644):415-9. Erratum in: Science. 2003 Oct 24;302(5645):568.

17.

Murine leukemias with retroviral insertions at Lmo2 are predictive of the leukemias induced in SCID-X1 patients following retroviral gene therapy.

Davé UP, Akagi K, Tripathi R, Cleveland SM, Thompson MA, Yi M, Stephens R, Downing JR, Jenkins NA, Copeland NG.

PLoS Genet. 2009 May;5(5):e1000491. doi: 10.1371/journal.pgen.1000491. Epub 2009 May 22.

18.

gamma-c gene transfer into SCID X1 patients' B-cell lines restores normal high-affinity interleukin-2 receptor expression and function.

Hacein-Bey H, Cavazzana-Calvo M, Le Deist F, Dautry-Varsat A, Hivroz C, Rivière I, Danos O, Heard JM, Sugamura K, Fischer A, De Saint Basile G.

Blood. 1996 Apr 15;87(8):3108-16.

19.

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
20.

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1.

Hacein-Bey-Abina S, Garrigue A, Wang GP, Soulier J, Lim A, Morillon E, Clappier E, Caccavelli L, Delabesse E, Beldjord K, Asnafi V, MacIntyre E, Dal Cortivo L, Radford I, Brousse N, Sigaux F, Moshous D, Hauer J, Borkhardt A, Belohradsky BH, Wintergerst U, Velez MC, Leiva L, Sorensen R, Wulffraat N, Blanche S, Bushman FD, Fischer A, Cavazzana-Calvo M.

J Clin Invest. 2008 Sep;118(9):3132-42. doi: 10.1172/JCI35700.

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