β-Globin LCR and Intron Elements Cooperate and Direct Spatial Reorganization for Gene Therapy

The Locus Control Region (LCR) requires intronic elements within β-globin transgenes to direct high level expression at all ectopic integration sites. However, these essential intronic elements cannot be transmitted through retrovirus vectors and their deletion may compromise the therapeutic potenti...

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Autori principali: Buzina, Alla, Lo, Mandy Y. M., Moffett, Angela, Hotta, Akitsu, Fussner, Eden, Bharadwaj, Rikki R., Pasceri, Peter, Garcia-Martinez, J. Victor, Bazett-Jones, David P., Ellis, James
Natura: Artigo
Lingua:Inglês
Pubblicazione: Public Library of Science 2008
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Research Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC2271131/
https://ncbi.nlm.nih.gov/pubmed/18404216
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pgen.1000051
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