Correction of the Enzymatic and Functional Deficits in a Model of Pompe Disease Using Adeno-associated Virus Vectors
Open Access
- 1 May 2002
- journal article
- Published by Elsevier in Molecular Therapy
- Vol. 5 (5) , 571-578
- https://doi.org/10.1006/mthe.2002.0580
Abstract
No abstract availableKeywords
This publication has 52 references indexed in Scilit:
- Sustained and Complete Phenotype Correction of Hemophilia B Mice Following Intramuscular Injection of AAV1 Serotype VectorsMolecular Therapy, 2001
- Morphological and Physiological Restorations of Hereditary Form of Dilated Cardiomyopathy by Somatic Gene TherapyBiochemical and Biophysical Research Communications, 2001
- The Use of Adeno-Associated Virus to Circumvent the Maturation-Dependent Viral Transduction of Muscle FibersHuman Gene Therapy, 2000
- Rescue of Skeletal Muscles of γ-Sarcoglycan- Deficient Mice with Adeno-Associated Virus-Mediated Gene TransferMolecular Therapy, 2000
- Recombinant adeno-associated virus purification using novel methods improves infectious titer and yieldGene Therapy, 1999
- rAAV vector-mediated sarcogylcan gene transfer in a hamster model for limb girdle muscular dystrophyGene Therapy, 1999
- Novel Tools for Production and Purification of Recombinant Adenoassociated Virus VectorsHuman Gene Therapy, 1998
- Recombinant Adeno-Associated Viral Vectors Mediate Long-Term Transgene Expression in MuscleHuman Gene Therapy, 1997
- Bone Marrow Transplantation for Glycogen Storage Disease Type II (Pompé's Disease)New England Journal of Medicine, 1986
- Uptake and stability of human and bovine acid α-glucosidase in cultured fibroblasts and skeletal muscle cells from glycogenosis type II patientsExperimental Cell Research, 1984