Research Topics
Species | Elena L AronovichSummaryAffiliation: University of Minnesota Country: USA Publications
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Detail Information
Publications
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...
