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Beta-globin LCR and intron elements cooperate and direct spatial reorganization for gene therapy.

The Locus Control Region (LCR) requires intronic elements within beta-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 pote...

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Principais autores: Alla Buzina, Mandy Y M Lo, Angela Moffett, Akitsu Hotta, Eden Fussner, Rikki R Bharadwaj, Peter Pasceri, J Victor Garcia-Martinez, David P Bazett-Jones, James Ellis
Format: Artigo
Sprog:Inglês
Udgivet: Public Library of Science (PLoS) 2008-04-01
Serier:PLoS Genetics
Online adgang:http://europepmc.org/articles/PMC2271131?pdf=render
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