Evasion of Immune Responses to Introduced Human Acid α-Glucosidase by Liver-Restricted Expression in Glycogen Storage Disease Type II
Open Access
- 1 November 2005
- journal article
- research article
- Published by Elsevier in Molecular Therapy
- Vol. 12 (5) , 876-884
- https://doi.org/10.1016/j.ymthe.2005.04.024
Abstract
No abstract availableKeywords
This publication has 46 references indexed in Scilit:
- Enumerating Antigen-Specific T-Cell Responses in Peripheral BloodJournal of Immunotherapy, 2005
- 867. Correction of Glycogen Storage Disease Type II by Intramuscular Administration of an Adeno-Associated Virus 6 (AAV2/6) Vector Containing a Muscle-Specific PromoterMolecular Therapy, 2004
- Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotypeBlood, 2004
- Phenotypic correction of a mouse model of hemophilia A using AAV2 vectors encoding the heavy and light chains of FVIIIBlood, 2003
- Packaging of an AAV vector encoding human acid α-glucosidase for gene therapy in glycogen storage disease type II with a modified hybrid adenovirus-AAV vectorMolecular Therapy, 2003
- Muscle-Specific Promoters May Be Necessary for Adeno-Associated Virus-Mediated Gene Transfer in the Treatment of Muscular DystrophiesHuman Gene Therapy, 2001
- Correction of Glycogen Storage Disease Type II by Enzyme Replacement with a Recombinant Human Acid Maltase Produced by Over-Expression in a CHO-DHFRneg Cell LineBiochemical and Biophysical Research Communications, 2000
- Enhanced Expression of Transgenes from Adeno-Associated Virus Vectors with the Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element: Implications for Gene TherapyHuman Gene Therapy, 1999
- Quantitation of a urinary tetrasaccharide by gas chromatography and mass spectrometryJournal of Mass Spectrometry, 1976
- Increased Excretion of a Glucose‐Containing Tetrasaccharide in the Urine of a Patient with Glycogen Storage Disease Type II (Pompe's Disease)European Journal of Clinical Investigation, 1974