Detalhe da pesquisa
1.
Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosis.
Mol Ther
; 22(8): 1472-1483, 2014 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-24869932
2.
Alterations in the adenosine metabolism and CD39/CD73 adenosinergic machinery cause loss of Treg cell function and autoimmunity in ADA-deficient SCID.
Blood
; 119(6): 1428-39, 2012 Feb 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-22184407
3.
ADA-deficient SCID is associated with a specific microenvironment and bone phenotype characterized by RANKL/OPG imbalance and osteoblast insufficiency.
Blood
; 114(15): 3216-26, 2009 Oct 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-19633200
4.
Conditioning Regimens in Long-Term Pre-Clinical Studies to Support Development of Ex Vivo Gene Therapy: Review of Nonproliferative and Proliferative Changes.
Hum Gene Ther
; 32(1-2): 66-76, 2021 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-32883113
5.
Lentiviral correction of enzymatic activity restrains macrophage inflammation in adenosine deaminase 2 deficiency.
Blood Adv
; 5(16): 3174-3187, 2021 08 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-34424322
6.
Bone marrow stromal cells from ß-thalassemia patients have impaired hematopoietic supportive capacity.
J Clin Invest
; 129(4): 1566-1580, 2019 02 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-30830876
7.
Good Laboratory Practice Preclinical Safety Studies for GSK2696273 (MLV Vector-Based Ex Vivo Gene Therapy for Adenosine Deaminase Deficiency Severe Combined Immunodeficiency) in NSG Mice.
Hum Gene Ther Clin Dev
; 28(1): 17-27, 2017 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-28319446
8.
Alterations in the brain adenosine metabolism cause behavioral and neurological impairment in ADA-deficient mice and patients.
Sci Rep
; 7: 40136, 2017 01 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-28074903
9.
Efficacy of gene therapy for Wiskott-Aldrich syndrome using a WAS promoter/cDNA-containing lentiviral vector and nonlethal irradiation.
Hum Gene Ther
; 17(3): 303-13, 2006 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-16544979
10.
Ex vivo gene therapy with lentiviral vectors rescues adenosine deaminase (ADA)-deficient mice and corrects their immune and metabolic defects.
Blood
; 108(9): 2979-88, 2006 Nov 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-16835374
11.
IL-3 or IL-7 increases ex vivo gene transfer efficiency in ADA-SCID BM CD34+ cells while maintaining in vivo lymphoid potential.
Mol Ther
; 10(6): 1096-108, 2004 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-15564141