Systemic Correction of Storage Disease in MPS I NOD/SCID Mice Using the Sleeping Beauty Transposon System
Open Access
- 1 July 2009
- journal article
- Published by Elsevier in Molecular Therapy
- Vol. 17 (7), 1136-1144
- https://doi.org/10.1038/mt.2009.87
Abstract
No abstract availableKeywords
This publication has 50 references indexed in Scilit:
- Sex-specific genetic architecture of human diseaseNature Reviews Genetics, 2008
- Improved retroviral vector design results in sustained expression after adult gene therapy in mucopolysaccharidosis I miceThe Journal of Gene Medicine, 2008
- Enhancing transduction of the liver by adeno-associated viral vectorsGene Therapy, 2008
- Chemically modified β-glucuronidase crosses blood–brain barrier and clears neuronal storage in murine mucopolysaccharidosis VIIProceedings of the National Academy of Sciences, 2008
- Preferential delivery of the Sleeping Beauty transposon system to livers of mice by hydrodynamic injectionNature Protocols, 2007
- Characterization of an immunodeficient mouse model of mucopolysaccharidosis type I suitable for preclinical testing of human stem cell and gene therapyBrain Research Bulletin, 2007
- Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon‐mediated gene delivery: implications for non‐viral gene therapy of mucopolysaccharidosesThe Journal of Gene Medicine, 2007
- Predicting preferential DNA vector insertion sites: implications for functional genomics and gene therapyGenome Biology, 2007
- Integrating DNA Vectors For Gene TherapyMolecular Therapy, 2007
- Occurrence of leukaemia following gene therapy of X-linked SCIDNature Reviews Cancer, 2003