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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...
Autores principales: | 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 |
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Formato: | Texto |
Lenguaje: | English |
Publicado: |
Public Library of Science
2008
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2271131/ https://www.ncbi.nlm.nih.gov/pubmed/18404216 http://dx.doi.org/10.1371/journal.pgen.1000051 |
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