Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter
Open Access
- 30 June 2005
- journal article
- Published by Elsevier in Molecular Therapy
- Vol. 11 (6) , 889-898
- https://doi.org/10.1016/j.ymthe.2005.01.012
Abstract
No abstract availableKeywords
This publication has 43 references indexed in Scilit:
- 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
- Influence of Vector Dose on Factor IX-Specific T and B Cell Responses in Muscle-Directed Gene TherapyHuman Gene Therapy, 2002
- Muscle-Directed Gene Transfer and Transient Immune Suppression Result in Sustained Partial Correction of Canine Hemophilia B Caused by a Null MutationMolecular Therapy, 2001
- Risk and Prevention of Anti-factor IX Formation in AAV-Mediated Gene Transfer in the Context of a Large Deletion of F9Molecular Therapy, 2001
- Purification of Recombinant Adeno-Associated Virus Vectors by Column Chromatography and Its Performancein VivoHuman Gene Therapy, 2000
- High-Titer Recombinant Adeno-Associated Virus Production from Replicating Amplicons and Herpes Vectors Deleted for Glycoprotein HHuman Gene Therapy, 1999
- Recombinant adeno-associated virus purification using novel methods improves infectious titer and yieldGene Therapy, 1999
- Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophiliaGene Therapy, 1998
- Isolation and characterization of packaging cell lines that coexpress the adenovirus E1, DNA polymerase, and preterminal proteins: implications for gene therapyGene Therapy, 1997
- Identification of a nonsense mutation in the carboxyl-terminal region of DNA-dependent protein kinase catalytic subunit in the scid mouse.Proceedings of the National Academy of Sciences, 1996