Detalhe da pesquisa
1.
Unregulated actin polymerization by WASp causes defects of mitosis and cytokinesis in X-linked neutropenia.
J Exp Med
; 204(9): 2213-24, 2007 Sep 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-17724125
2.
Codon optimization of human factor VIII cDNAs leads to high-level expression.
Blood
; 117(3): 798-807, 2011 Jan 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-21041718
3.
Effective gene therapy with nonintegrating lentiviral vectors.
Nat Med
; 12(3): 348-53, 2006 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-16491086
4.
Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells.
Mol Ther
; 19(1): 122-32, 2011 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-20978475
5.
Phosphorylation of WASp is a key regulator of activity and stability in vivo.
Proc Natl Acad Sci U S A
; 106(37): 15738-43, 2009 Sep 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-19805221
6.
Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients.
J Clin Invest
; 118(9): 3143-50, 2008 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-18688286
7.
Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo.
J Clin Invest
; 117(8): 2241-9, 2007 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-17671654
8.
The gene involved in X-linked agammaglobulinaemia is a member of the Src family of protein-tyrosine kinases. 1993.
J Immunol
; 188(7): 2948-55, 2012 Apr 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-22442492
9.
Improvement of migratory defects in a murine model of Wiskott-Aldrich syndrome gene therapy.
Mol Ther
; 16(5): 836-44, 2008 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-18388921
10.
Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency.
Mol Ther
; 16(3): 590-8, 2008 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-18180772
11.
Enhancement of mouse hematopoietic stem/progenitor cell function via transient gene delivery using integration-deficient lentiviral vectors.
Exp Hematol
; 57: 21-29, 2018 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-28911908
12.
Bone morphogenetic protein-4 (BMP4) up regulates key haematopoietic genes in differentiating embryonic stem cells treated with BMP4 short hairpin RNA.
Br J Haematol
; 155(5): 638-41, 2011 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-21749358
13.
Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector.
Lancet
; 364(9452): 2181-7, 2004.
Artigo
em Inglês
| MEDLINE | ID: mdl-15610804
14.
High-level transduction and gene expression in hematopoietic repopulating cells using a human immunodeficiency [correction of imunodeficiency] virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter.
Hum Gene Ther
; 13(7): 803-13, 2002 May 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-11975847
15.
Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency.
Sci Transl Med
; 3(97): 97ra79, 2011 Aug 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-21865537
16.
Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction.
Sci Transl Med
; 3(97): 97ra80, 2011 Aug 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-21865538
17.
Cyclooxygenase-2 overexpression, using an integrin-targeted gene delivery system (the LID vector), inhibits fibroblast proliferation in vitro and leads to increased prostaglandin E(2) in the lung.
Chest
; 121(3 Suppl): 102S-104S, 2002 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-11893724
18.
Gene therapy of inherited immunodeficiencies.
Expert Opin Biol Ther
; 8(4): 397-407, 2008 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-18352845
19.
Bone morphogenetic protein 4 modulates c-Kit expression and differentiation potential in murine embryonic aorta-gonad-mesonephros haematopoiesis in vitro.
Br J Haematol
; 139(2): 321-30, 2007 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-17897310
20.
Lentiviral vectors for T-cell suicide gene therapy: preservation of T-cell effector function after cytokine-mediated transduction.
Mol Ther
; 15(2): 355-60, 2007 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-17235314