Detalhe da pesquisa
1.
Unbiased assessment of genome integrity and purging of adverse outcomes at the target locus upon editing of CD4+ T-cells for the treatment of Hyper IgM1.
EMBO J
; 42(23): e114188, 2023 Dec 01.
Artigo
Inglês
| MEDLINE | ID: mdl-37916874
2.
Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy.
Mol Ther
; 29(1): 86-102, 2021 01 06.
Artigo
Inglês
| MEDLINE | ID: mdl-33010230
3.
The galactocerebrosidase enzyme contributes to the maintenance of a functional hematopoietic stem cell niche.
Blood
; 116(11): 1857-66, 2010 Sep 16.
Artigo
Inglês
| MEDLINE | ID: mdl-20511539
4.
Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model.
Blood
; 116(24): 5130-9, 2010 Dec 09.
Artigo
Inglês
| MEDLINE | ID: mdl-20847202
5.
Maintenance of a functional hematopoietic stem cell niche through galactocerebrosidase and other enzymes.
Curr Opin Hematol
; 18(4): 214-9, 2011 Jul.
Artigo
Inglês
| MEDLINE | ID: mdl-21537167
6.
Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates.
Nat Commun
; 13(1): 2454, 2022 05 04.
Artigo
Inglês
| MEDLINE | ID: mdl-35508619
7.
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
Inglês
| MEDLINE | ID: mdl-32883113
8.
Dlg1, Sec8, and Mtmr2 regulate membrane homeostasis in Schwann cell myelination.
J Neurosci
; 29(27): 8858-70, 2009 Jul 08.
Artigo
Inglês
| MEDLINE | ID: mdl-19587293
9.
Development and maturation of invariant NKT cells in the presence of lysosomal engulfment.
Eur J Immunol
; 39(10): 2748-54, 2009 Oct.
Artigo
Inglês
| MEDLINE | ID: mdl-19637231
10.
Monitoring disease evolution and treatment response in lysosomal disorders by the peripheral benzodiazepine receptor ligand PK11195.
Neurobiol Dis
; 34(1): 51-62, 2009 Apr.
Artigo
Inglês
| MEDLINE | ID: mdl-19320046
11.
Specific determination of beta-galactocerebrosidase activity via competitive inhibition of beta-galactosidase.
Clin Chem
; 55(3): 541-8, 2009 Mar.
Artigo
Inglês
| MEDLINE | ID: mdl-19147730
12.
Phagocytosis-shielded lentiviral vectors improve liver gene therapy in nonhuman primates.
Sci Transl Med
; 11(493)2019 05 22.
Artigo
Inglês
| MEDLINE | ID: mdl-31118293
13.
SUMF1 enhances sulfatase activities in vivo in five sulfatase deficiencies.
Biochem J
; 403(2): 305-12, 2007 Apr 15.
Artigo
Inglês
| MEDLINE | ID: mdl-17206939
14.
Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells.
J Clin Invest
; 113(8): 1118-29, 2004 Apr.
Artigo
Inglês
| MEDLINE | ID: mdl-15085191
15.
Preclinical Testing of the Safety and Tolerability of Lentiviral Vector-Mediated Above-Normal Alpha-L-Iduronidase Expression in Murine and Human Hematopoietic Cells Using Toxicology and Biodistribution Good Laboratory Practice Studies.
Hum Gene Ther
; 27(10): 813-829, 2016 10.
Artigo
Inglês
| MEDLINE | ID: mdl-27431943
16.
Sulfatase modifying factor 1-mediated fibroblast growth factor signaling primes hematopoietic multilineage development.
J Exp Med
; 207(8): 1647-60, 2010 Aug 02.
Artigo
Inglês
| MEDLINE | ID: mdl-20643830
17.
Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy.
Sci Transl Med
; 2(58): 58ra84, 2010 Nov 17.
Artigo
Inglês
| MEDLINE | ID: mdl-21084719