Systemic Correction of Storage Disease in MPS I NOD/SCID Mice Using the Sleeping Beauty Transposon System

The Sleeping Beauty (SB) transposon system is a nonviral vector that directs transgene integration into vertebrate genomes. We hydrodynamically delivered SB transposon plasmids encoding human α-L-iduronidase (hIDUA) at two DNA doses, with and without an SB transposase gene, to NOD.129(B6)-Prkdc(scid...

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Detalhes bibliográficos
Main Authors: Aronovich, Elena L, Bell, Jason B, Khan, Shaukat A, Belur, Lalitha R, Gunther, Roland, Koniar, Brenda, Schachern, Patricia A, Parker, Josh B, Carlson, Cathy S, Whitley, Chester B, McIvor, R Scott, Gupta, Pankaj, Hackett, Perry B
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2009
Assuntos:
Original Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2835207/
https://ncbi.nlm.nih.gov/pubmed/19384290
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.87
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