Detalhe da pesquisa
1.
Laminin-521 Protein Therapy for Glomerular Basement Membrane and Podocyte Abnormalities in a Model of Pierson Syndrome.
J Am Soc Nephrol
; 29(5): 1426-1436, 2018 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-29472414
2.
Laminin-111 improves muscle repair in a mouse model of merosin-deficient congenital muscular dystrophy.
Hum Mol Genet
; 23(2): 383-96, 2014 Jan 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-24009313
3.
Laminin-111 protein therapy reduces muscle pathology and improves viability of a mouse model of merosin-deficient congenital muscular dystrophy.
Am J Pathol
; 180(4): 1593-602, 2012 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-22322301
4.
Systemic administration of AAV8-α-galactosidase A induces humoral tolerance in nonhuman primates despite low hepatic expression.
Mol Ther
; 19(11): 1999-2011, 2011 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-21712814
5.
Targeting Pathogenic Lafora Bodies in Lafora Disease Using an Antibody-Enzyme Fusion.
Cell Metab
; 30(4): 689-705.e6, 2019 10 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-31353261
6.
Cell and gene-based therapies for the lysosomal storage diseases.
Curr Gene Ther
; 6(2): 227-41, 2006 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-16611044
7.
Development of catheter-based procedures for transducing the isolated rabbit liver with plasmid DNA.
Hum Gene Ther
; 13(17): 2065-77, 2002 Nov 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-12490001
8.
Hydrodynamic delivery of DNA.
Expert Opin Biol Ther
; 3(6): 911-8, 2003 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-12943450
9.
Ability of adeno-associated virus serotype 8-mediated hepatic expression of acid alpha-glucosidase to correct the biochemical and motor function deficits of presymptomatic and symptomatic Pompe mice.
Hum Gene Ther
; 19(6): 609-21, 2008 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-18500944
10.
Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease.
Mol Ther
; 15(10): 1782-8, 2007 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-17637720
11.
Local delivery of a viral vector mitigates neutralization by antiviral antibodies and results in efficient transduction of rabbit liver.
Mol Ther
; 12(6): 1043-51, 2005 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-16139571
12.
Long-term transgene expression from plasmid DNA gene therapy vectors is negatively affected by CpG dinucleotides.
Mol Ther
; 10(2): 269-78, 2004 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-15294174
13.
Efficacy of gene therapy for a prototypical lysosomal storage disease (GSD-II) is critically dependent on vector dose, transgene promoter, and the tissues targeted for vector transduction.
Mol Ther
; 5(4): 436-46, 2002 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-11945071
14.
Acute regression of advanced and retardation of early aortic atheroma in immunocompetent apolipoprotein-E (apoE) deficient mice by administration of a second generation [E1(-), E3(-), polymerase(-)] adenovirus vector expressing human apoE.
Hum Mol Genet
; 11(1): 43-58, 2002 Jan 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-11772998