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Restoration of lymphocyte function in Janus kinase 3-deficient mice by retroviral-mediated gene transfer
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Bunting KD, Sangster MY, Ihle JN, Sorrentino BP: Restoration of lymphocyte function in Janus kinase 3-deficient mice by retroviral-mediated gene transfer. Nat Med 1998, 4:58-64. The authors demonstrated that retroviral-mediated JAK3 gene transfer can reconstitute normal immune system in JAK3-deficient mice, which is one of the convincing successes of gene therapy models for congenital genetic disorders.
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Bunting, K.D.1
Sangster, M.Y.2
Ihle, J.N.3
Sorrentino, B.P.4
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44
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0033524390
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Virus-specific immunity after gene therapy in a murine model of severe combined immunodeficiency
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Bunting KD, Flynn KJ, Riberdy JM, Doherty PC, Sorrentino BP: Virus-specific immunity after gene therapy in a murine model of severe combined immunodeficiency. Proc Natl Acad Sci U S A 1999, 96:232-237.
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Bunting, K.D.1
Flynn, K.J.2
Riberdy, J.M.3
Doherty, P.C.4
Sorrentino, B.P.5
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45
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0031787859
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Thymic lymphoproliferative disease after successful correction of CD40 ligand deficiency by gene transfer in mice
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Brown MP, Topham DJ, Sangster MY, Zhao J, Flynn KJ, Surman SL, et al.: Thymic lymphoproliferative disease after successful correction of CD40 ligand deficiency by gene transfer in mice. Nat Med 1998, 4:1253-1260. This study showed that constitutive expression of CD40 ligand induced by a retroviral vector can improve immune function in CD40 ligand-deficient mice. However, more than 60% of treated mice developed T-cell lymphoproliferative disorders, suggesting current retroviral gene therapy is not adequate for genes that require tight regulation of expression.
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Brown, M.P.1
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Sangster, M.Y.3
Zhao, J.4
Flynn, K.J.5
Surman, S.L.6
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46
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0030667287
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Btk dosage determines sensitivity to B cell antigen receptor cross-linking
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Satterthwaite AB, Cheroutre H, Khan WN, Sideras P, Witte ON: Btk dosage determines sensitivity to B cell antigen receptor cross-linking. Proc Natl Acad Sci U S A 1997, 94:13152-13157.
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Cheroutre, H.2
Khan, W.N.3
Sideras, P.4
Witte, O.N.5
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47
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0030758466
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The X-linked immunodeficiency defect in the mouse is corrected by expression of human Bruton's tyrosine kinase from a yeast artificial chromosome transgene
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Maas A, Dingjan GM, Savelkoul HF, Kinnon C, Grosveld F, Hendriks RW: The X-linked immunodeficiency defect in the mouse is corrected by expression of human Bruton's tyrosine kinase from a yeast artificial chromosome transgene. Eur J Immunol 1997, 27:2180-2187.
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Maas, A.1
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Kinnon, C.4
Grosveld, F.5
Hendriks, R.W.6
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48
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0031025342
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Correction of the X-linked immunodeficiency phenotype by transgenic expression of human Bruton's tyrosine kinase under the control of the class II major histocompatibility complex Ea locus control region
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Drabek D, Raguz S, De Wit TP, Dingjan GM, Savelkoul HF, Grosveld F, et al.: Correction of the X-linked immunodeficiency phenotype by transgenic expression of human Bruton's tyrosine kinase under the control of the class II major histocompatibility complex Ea locus control region. Proc Natl Acad Sci U S A 1997, 94:610-615.
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Dingjan, G.M.4
Savelkoul, H.F.5
Grosveld, F.6
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49
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Correction of X-linked immunodeficient mice by competitive reconstitution with limiting numbers of normal bone marrow cells
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in press
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Rohrer J, Conley ME: Correction of X-linked immunodeficient mice by competitive reconstitution with limiting numbers of normal bone marrow cells. Blood 1999, in press. This paper shows that limited numbers of normal cells are sufficient for the correction of humoral immunity in Btk-deficient mice using competitive bone marrow transplantation.
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Blood
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Rohrer, J.1
Conley, M.E.2
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