Fully Deleted Adenovirus Persistently Expressing GAA Accomplishes Long-Term Skeletal Muscle Glycogen Correction in Tolerant and Nontolerant GSD-II Mice
Open Access
- 1 January 2006
- journal article
- research article
- Published by Elsevier in Molecular Therapy
- Vol. 13 (1) , 127-134
- https://doi.org/10.1016/j.ymthe.2005.08.006
Abstract
No abstract availableKeywords
This publication has 33 references indexed in Scilit:
- Primary adenovirus-specific cytotoxic T lymphocyte response occurs after viral clearance and liver enzyme elevationGene Therapy, 2005
- Multiple muscles in the AMD quail can be ?cross-corrected? of pathologic glycogen accumulation after intravenous injection of an [E1-, polymerase-] adenovirus vector encoding human acid-?-glucosidaseThe Journal of Gene Medicine, 2002
- Efficacy of Gene Therapy for a Prototypical Lysosomal Storage Disease (GSD-II) Is Critically Dependent on Vector Dose, Transgene Promoter, and the Tissues Targeted for Vector TransductionMolecular Therapy, 2002
- Long-Term Efficacy after [E1-, polymerase-] Adenovirus-Mediated Transfer of Human Acid-α-Glucosidase Gene into Glycogen Storage Disease Type II Knockout MiceHuman Gene Therapy, 2001
- Towards a molecular therapy for glycogen storage disease type II (Pompe disease)Molecular Medicine Today, 2000
- Frequency of glycogen storage disease type II in The Netherlands: implications for diagnosis and genetic counsellingEuropean Journal of Human Genetics, 1999
- Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus vector encoding human acid-α-glucosidaseProceedings of the National Academy of Sciences, 1999
- A diagnostic protocol for adult-onset glycogen storage disease type IINeurology, 1999
- Persistence of an [E1-, Polymerase-] Adenovirus Vector Despite Transduction of a Neoantigen into Immune-Competent MiceHuman Gene Therapy, 1999
- Carrier frequency for glycogen storage disease type II in New York and estimates of affected individuals born with the diseaseAmerican Journal of Medical Genetics, 1998