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Priapism in a Fabry disease mouse model is associated with upregulated penile nNOS and eNOS expression.
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- Journal of Inherited Metabolic Disease, 2018, v. 41, n. 2, p. 231, doi. 10.1007/s10545-017-0107-6
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- Article
Adeno-associated virus-mediated expression of β-hexosaminidase prevents neuronal loss in the Sandhoff mouse brain.
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- Human Molecular Genetics, 2011, v. 20, n. 22, p. 4371, doi. 10.1093/hmg/ddr364
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- Article
Partial correction of the α-galactosidase A deficiency and reduction of glycolipid storage in Fabry mice using synthetic vectors.
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- Journal of Gene Medicine, 2004, v. 6, n. 1, p. 85, doi. 10.1002/jgm.468
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- Article
Systemic Administration of AAV8-α-galactosidase A Induces Humoral Tolerance in Nonhuman Primates Despite Low Hepatic Expression.
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- Molecular Therapy, 2011, v. 19, n. 11, p. 1999, doi. 10.1038/mt.2011.119
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- Article
Preexisting Immunity and Low Expression in Primates Highlight Translational Challenges for Liver-directed AAV8-mediated Gene Therapy.
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- Molecular Therapy, 2010, v. 18, n. 11, p. 1983, doi. 10.1038/mt.2010.175
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- Article
Evaluation of Systemic Follistatin as an Adjuvant to Stimulate Muscle Repair and Improve Motor Function in Pompe Mice.
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- Molecular Therapy, 2010, v. 18, n. 9, p. 1584, doi. 10.1038/mt.2010.110
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- Article
Glycoengineered Acid α-Glucosidase With Improved Efficacy at Correcting the Metabolic Aberrations and Motor Function Deficits in a Mouse Model of Pompe Disease.
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- Molecular Therapy, 2009, v. 17, n. 6, p. 954, doi. 10.1038/mt.2009.37
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- Article
Correction of the Biochemical and Functional Deficits in Fabry Mice Following AAV8–mediated Hepatic Expression of α-galactosidase A.
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- Molecular Therapy, 2007, v. 15, n. 3, p. 492, doi. 10.1038/sj.mt.6300066
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- Article
217. Comparison of Hepatic Vein and Peripheral Vein Delivery of an AAV2/8 Vector Encoding Human a-Galactosidase A to Rhesus Macaques.
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- Molecular Therapy, 2006, v. 13, p. S84, doi. 10.1016/j.ymthe.2006.08.242
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- Article
417. Combination Brain and Systemic Injections of AAV Results in Whole Body Therapy and Extension of Lifespan in the Niemann-Pick Mouse.
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- Molecular Therapy, 2006, v. 13, p. S160, doi. 10.1016/j.ymthe.2006.08.481
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- Article
AAV8-Mediated Hepatic Expression of Acid Sphingomyelinase Corrects the Metabolic Defect in the Visceral Organs of a Mouse Model of Niemann–Pick Disease
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- Molecular Therapy, 2005, v. 12, n. 3, p. 431, doi. 10.1016/j.ymthe.2005.03.011
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- Article
AAV2 Vector Harboring a Liver-Restricted Promoter Facilitates Sustained Expression of Therapeutic Levels of α-Galactosidase A and the Induction of Immune Tolerance in Fabry Mice
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- Molecular Therapy, 2004, v. 9, n. 2, p. 231, doi. 10.1016/j.ymthe.2003.11.015
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- Article
Demonstration of Feasibility of In Vivo Gene Therapy for Gaucher Disease Using a Chemically Induced Mouse Model
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- Molecular Therapy, 2002, v. 6, n. 2, p. 179, doi. 10.1006/mthe.2002.0650
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- Article
Adenovirus-Transduced Lung as a Portal for Delivering α-Galactosidase A into Systemic Circulation for Fabry Disease
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- Molecular Therapy, 2002, v. 5, n. 6, p. 745, doi. 10.1006/mthe.2002.0605
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- Article