Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses

BACKGROUND: 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. METHODS: We constructed SB transposon plasmids f...

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Auteurs principaux: Aronovich, Elena L., Bell, Jason B., Belur, Lalitha R., Gunther, Roland, Koniar, Brenda, Erickson, David C. C., Schachern, Patricia A., Matise, Ilze, McIvor, R. Scott, Whitley, Chester B., Hackett, Perry B.
Format: Artigo
Langue:Inglês
Publié: 2007
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Article
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC1868578/
https://ncbi.nlm.nih.gov/pubmed/17407189
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jgm.1028
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