Detalhe da pesquisa
1.
T-cell gene therapy for perforin deficiency corrects cytotoxicity defects and prevents hemophagocytic lymphohistiocytosis manifestations.
J Allergy Clin Immunol
; 142(3): 904-913.e3, 2018 09.
Artigo
Inglês
| MEDLINE | ID: mdl-29355678
2.
Mutations in linker for activation of T cells (LAT) lead to a novel form of severe combined immunodeficiency.
J Allergy Clin Immunol
; 139(2): 634-642.e5, 2017 02.
Artigo
Inglês
| MEDLINE | ID: mdl-27522155
3.
Perforin gene transfer into hematopoietic stem cells improves immune dysregulation in murine models of perforin deficiency.
Mol Ther
; 23(4): 737-45, 2015 Apr.
Artigo
Inglês
| MEDLINE | ID: mdl-25523759
4.
Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency.
Mol Ther
; 22(3): 607-622, 2014 Mar.
Artigo
Inglês
| MEDLINE | ID: mdl-24256635
5.
The ß-globin locus control region in combination with the EF1α short promoter allows enhanced lentiviral vector-mediated erythroid gene expression with conserved multilineage activity.
Mol Ther
; 20(7): 1400-9, 2012 Jul.
Artigo
Inglês
| MEDLINE | ID: mdl-22434141
6.
Identification of a small molecule for enhancing lentiviral transduction of T cells.
Mol Ther Methods Clin Dev
; 31: 101113, 2023 Dec 14.
Artigo
Inglês
| MEDLINE | ID: mdl-37790244
7.
Rapid Lentiviral Vector Producer Cell Line Generation Using a Single DNA Construct.
Mol Ther Methods Clin Dev
; 19: 47-57, 2020 Dec 11.
Artigo
Inglês
| MEDLINE | ID: mdl-32995359
8.
Stabilization of gammaretroviral and lentiviral vectors: from production to gene transfer.
J Gene Med
; 11(8): 670-8, 2009 Aug.
Artigo
Inglês
| MEDLINE | ID: mdl-19507176
9.
Screening of novel excipients for improving the stability of retroviral and adenoviral vectors.
Biotechnol Prog
; 22(2): 568-76, 2006.
Artigo
Inglês
| MEDLINE | ID: mdl-16599578
10.
High Level of Perforin Expression Is Required for Effective Correction of Hemophagocytic Lymphohistiocytosis.
Hum Gene Ther
; 27(10): 847-859, 2016 10.
Artigo
Inglês
| MEDLINE | ID: mdl-27471778
11.
Gene therapy for haemophagocytic lymphohistiocytosis.
Curr Gene Ther
; 14(6): 437-46, 2014.
Artigo
Inglês
| MEDLINE | ID: mdl-25245087
12.
Manufacturing of retroviruses.
Methods Mol Biol
; 737: 157-82, 2011.
Artigo
Inglês
| MEDLINE | ID: mdl-21590397
13.
Scaleable purification process for gene therapy retroviral vectors.
J Gene Med
; 9(4): 233-43, 2007 Apr.
Artigo
Inglês
| MEDLINE | ID: mdl-17428003