Found: 8
Select item for more details and to access through your institution.
Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry Disease.
- Published in:
- Molecular Medicine, 2015, v. 21, n. 1, p. 389, doi. 10.2119/molmed.2015.00088
- By:
- Publication type:
- Article
AAV8-mediated expression of glucocerebrosidase ameliorates the storage pathology in the visceral organs of a mouse model of Gaucher disease.
- Published in:
- Journal of Gene Medicine, 2006, v. 8, n. 6, p. 719, doi. 10.1002/jgm.901
- By:
- Publication type:
- Article
Systemic Administration of AAV8-α-galactosidase A Induces Humoral Tolerance in Nonhuman Primates Despite Low Hepatic Expression.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 11, p. 1999, doi. 10.1038/mt.2011.119
- By:
- Publication type:
- Article
Preexisting Immunity and Low Expression in Primates Highlight Translational Challenges for Liver-directed AAV8-mediated Gene Therapy.
- Published in:
- Molecular Therapy, 2010, v. 18, n. 11, p. 1983, doi. 10.1038/mt.2010.175
- By:
- Publication type:
- Article
217. Comparison of Hepatic Vein and Peripheral Vein Delivery of an AAV2/8 Vector Encoding Human a-Galactosidase A to Rhesus Macaques.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S84, doi. 10.1016/j.ymthe.2006.08.242
- By:
- Publication type:
- Article
910. AAV8-Mediated Expression of Glucocerebrosidase Ameliorates the Storage Pathology in the D409V/null Gaucher Mouse
- Published in:
- 2005
- By:
- Publication type:
- Abstract
Demonstration of Feasibility of In Vivo Gene Therapy for Gaucher Disease Using a Chemically Induced Mouse Model
- Published in:
- Molecular Therapy, 2002, v. 6, n. 2, p. 179, doi. 10.1006/mthe.2002.0650
- By:
- Publication type:
- Article
Iminosugar-Based Inhibitors of Glucosylceramide Synthase Increase Brain Glycosphingolipids and Survival in a Mouse Model of Sandhoff Disease.
- Published in:
- PLoS ONE, 2011, v. 6, n. 6, p. 1, doi. 10.1371/journal.pone.0021758
- By:
- Publication type:
- Article