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1.
Priapism in a Fabry disease mouse model is associated with upregulated penile nNOS and eNOS expression.
J Inherit Metab Dis
; 41(2): 231-238, 2018 03.
Artículo
en Inglés
| MEDLINE | ID: mdl-29110178
2.
Adeno-associated virus-mediated expression of ß-hexosaminidase prevents neuronal loss in the Sandhoff mouse brain.
Hum Mol Genet
; 20(22): 4371-80, 2011 Nov 15.
Artículo
en Inglés
| MEDLINE | ID: mdl-21852247
3.
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.
Artículo
en Inglés
| MEDLINE | ID: mdl-21712814
4.
Evaluation of systemic follistatin as an adjuvant to stimulate muscle repair and improve motor function in Pompe mice.
Mol Ther
; 18(9): 1584-91, 2010 Sep.
Artículo
en Inglés
| MEDLINE | ID: mdl-20551907
5.
Preexisting immunity and low expression in primates highlight translational challenges for liver-directed AAV8-mediated gene therapy.
Mol Ther
; 18(11): 1983-94, 2010 Nov.
Artículo
en Inglés
| MEDLINE | ID: mdl-20736932
6.
Glycoengineered acid alpha-glucosidase with improved efficacy at correcting the metabolic aberrations and motor function deficits in a mouse model of Pompe disease.
Mol Ther
; 17(6): 954-63, 2009 Jun.
Artículo
en Inglés
| MEDLINE | ID: mdl-19277015
7.
Pulmonary delivery of recombinant acid sphingomyelinase improves clearance of lysosomal sphingomyelin from the lungs of a murine model of Niemann-Pick disease.
Mol Genet Metab
; 97(1): 35-42, 2009 May.
Artículo
en Inglés
| MEDLINE | ID: mdl-19231265
8.
Correction of the Biochemical and Functional Deficits in Fabry Mice Following AAV8-mediated Hepatic Expression of α-galactosidase A.
Mol Ther
; 15(3): 492-500, 2007 Mar.
Artículo
en Inglés
| MEDLINE | ID: mdl-28182896
9.
Correction of the biochemical and functional deficits in fabry mice following AAV8-mediated hepatic expression of alpha-galactosidase A.
Mol Ther
; 15(3): 492-500, 2007 Mar.
Artículo
en Inglés
| MEDLINE | ID: mdl-17191071
10.
Correction of the nonlinear dose response improves the viability of adenoviral vectors for gene therapy of Fabry disease.
Hum Gene Ther
; 13(8): 935-45, 2002 May 20.
Artículo
en Inglés
| MEDLINE | ID: mdl-12031126
11.
Distribution of acid sphingomyelinase in rodent and non-human primate brain after intracerebroventricular infusion.
Exp Neurol
; 231(2): 261-71, 2011 Oct.
Artículo
en Inglés
| MEDLINE | ID: mdl-21777586
12.
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.
Artículo
en Inglés
| MEDLINE | ID: mdl-18500944
13.
Combination brain and systemic injections of AAV provide maximal functional and survival benefits in the Niemann-Pick mouse.
Proc Natl Acad Sci U S A
; 104(22): 9505-10, 2007 May 29.
Artículo
en Inglés
| MEDLINE | ID: mdl-17517638
14.
AAV8-mediated hepatic expression of acid sphingomyelinase corrects the metabolic defect in the visceral organs of a mouse model of Niemann-Pick disease.
Mol Ther
; 12(3): 431-40, 2005 Sep.
Artículo
en Inglés
| MEDLINE | ID: mdl-16099409
15.
Adenovirus-transduced lung as a portal for delivering alpha-galactosidase A into systemic circulation for Fabry disease.
Mol Ther
; 5(6): 745-54, 2002 Jun.
Artículo
en Inglés
| MEDLINE | ID: mdl-12027559
16.
Partial correction of the alpha-galactosidase A deficiency and reduction of glycolipid storage in Fabry mice using synthetic vectors.
J Gene Med
; 6(1): 85-92, 2004 Jan.
Artículo
en Inglés
| MEDLINE | ID: mdl-14716680
17.
Demonstration of feasibility of in vivo gene therapy for Gaucher disease using a chemically induced mouse model.
Mol Ther
; 6(2): 179-89, 2002 Aug.
Artículo
en Inglés
| MEDLINE | ID: mdl-12161184
18.
AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice.
Mol Ther
; 9(2): 231-40, 2004 Feb.
Artículo
en Inglés
| MEDLINE | ID: mdl-14759807
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