Elena L Aronovich
Affiliation: University of Minnesota
- Systemic correction of storage disease in MPS I NOD/SCID mice using the sleeping beauty transposon systemElena L Aronovich
Department of Genetics, Cell Biology and Development, Center for Genome Engineering, University of Minnesota, Minneapolis, 55455, USA
Mol Ther 17:1136-44. 2009....
- The Sleeping Beauty transposon system: a non-viral vector for gene therapyElena L Aronovich
Department of Genetics, Cell Biology and Development, The Center for Genome Engineering, Institute of Human Genetics, University of Minnesota, 6 160 Jackson Hall, 321 Church St SE, Minneapolis, MN 55455, USA
Hum Mol Genet 20:R14-20. 2011..Progress in delivery of SB transposons to liver for treatment of various systemic diseases, such as hemophilia and mucopolysaccharidoses types I and VII, has encountered some problems, but even here progress is being made...
- Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidosesElena L Aronovich
Department of Genetics, Cell Biology and Development and The Arnold and Mabel Beckman Center for Transposon Research, University of Minnesota, Minneapolis, MN 55455, USA
J Gene Med 9:403-15. 2007..The Sleeping Beauty (SB) transposon system is a non-viral vector system that can integrate precise sequences into chromosomes. We evaluated the SB transposon system as a tool for gene therapy of mucopolysaccharidosis (MPS) types I and VII...
- Preferential delivery of the Sleeping Beauty transposon system to livers of mice by hydrodynamic injectionJason B Bell
Department of Genetics, Cell Biology and Development, Beckman Center for Transposon Research, Institute of Human Genetics, University of Minnesota, 6 160 Jackson Hall, 321 Church Street SE, Minneapolis, Minnesota 55455, USA
Nat Protoc 2:3153-65. 2007..Several weeks after injection, transgene expression stabilizes at approximately 1% of the level at 24 h, presumably owing to integration of the transposons into chromosomes...
- Efficacy and safety of Sleeping Beauty transposon-mediated gene transfer in preclinical animal studiesPerry B Hackett
Dept of Genetics, Cell Biology and Development, 321 Church St SE, Minneapolis, MN 55455, USA
Curr Gene Ther 11:341-9. 2011..The ability to correct genetic diseases through the infusion of DNA plasmids remains an appealing goal...
- Excision of Sleeping Beauty transposons: parameters and applications to gene therapyGeyi Liu
Department of Genetics, Cell Biology and Development and The Institute of Human Genetics, University of Minnesota, Minneapolis, MN 55455, USA
J Gene Med 6:574-83. 2004..The excision assay appears to be a relatively quick and easy method to optimize protocols for delivery of genes in SB transposons to mammalian chromosomes in living animals...
- Duration of expression and activity of Sleeping Beauty transposase in mouse liver following hydrodynamic DNA deliveryJason B Bell
Department of Genetics, University of Minnesota, Minneapolis, Minnesota, USA
Mol Ther 18:1796-802. 2010..Thus, within the limits of current technology, we show that SB transposons appear to be as stably integrated as their viral counterparts...
- Lysosomal enzyme can bypass the blood-brain barrier and reach the CNS following intranasal administrationDaniel A Wolf
Gene Therapy Program, Institute of Human Genetics, Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis MN 55455, USA
Mol Genet Metab 106:131-4. 2012..These results suggest that intranasal routes of delivery may be efficacious in the treatment of lysosomal storage disorders...
- Sleeping Beauty-mediated correction of Fanconi anemia type CKendra A Hyland
Discovery Genomics, Inc, Minneapolis, MN, USA
J Gene Med 13:462-9. 2011..Our goal is to develop the SB system for nonviral complementation of Fanconi anemia (FA), a rare autosomal recessive disorder accompanied by progressive bone marrow failure...