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
Open Access
- 1 February 2004
- journal article
- research article
- Published by Elsevier in Molecular Therapy
- Vol. 9 (2) , 231-240
- https://doi.org/10.1016/j.ymthe.2003.11.015
Abstract
No abstract availableKeywords
This publication has 38 references indexed in Scilit:
- Long-term correction of globotriaosylceramide storage in Fabry mice by recombinant adeno-associated virus-mediated gene transferProceedings of the National Academy of Sciences, 2003
- Long-term systemic therapy of Fabry disease in a knockout mouse by adeno-associated virus-mediated muscle-directed gene transferProceedings of the National Academy of Sciences, 2002
- Adenovirus-Transduced Lung as a Portal for Delivering α-Galactosidase A into Systemic Circulation for Fabry DiseaseMolecular Therapy, 2002
- Anderson–Fabry disease: Clinical manifestations of disease in female heterozygotesJournal of Inherited Metabolic Disease, 2001
- Safety and Efficacy of Recombinant Human α-Galactosidase A Replacement Therapy in Fabry's DiseaseNew England Journal of Medicine, 2001
- Enzyme Replacement Therapy in Fabry DiseaseJAMA, 2001
- Fabry Disease: Preclinical Studies Demonstrate the Effectiveness of α-Galactosidase A Replacement in Enzyme-Deficient MiceAmerican Journal of Human Genetics, 2001
- Correction of Enzymatic and Lysosomal Storage Defects in Fabry Mice by Adenovirus-Mediated Gene TransferHuman Gene Therapy, 1999
- Correction in trans for Fabry disease: expression, secretion and uptake of alpha-galactosidase A in patient-derived cells driven by a high-titer recombinant retroviral vector.Proceedings of the National Academy of Sciences, 1996
- LYSOSOMAL STORAGE DISEASESAnnual Review of Biochemistry, 1991