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Regulated high level expression of a human gamma-globin gene introduced into erythroid cells by an adeno-associated virus vector.

Gene therapy of severe hemoglobinopathies will require high-level expression of a transferred globin gene in erythroid cells. Distant regulatory elements flanking the beta-globin gene cluster, the locus control region, are needed for appropriate expression. We have explored the use of a human parvov...

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Autori principali: Walsh, C E, Liu, J M, Xiao, X, Young, N S, Nienhuis, A W, Samulski, R J
Natura: Artigo
Lingua:Inglês
Pubblicazione: 1992
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC49685/
https://ncbi.nlm.nih.gov/pubmed/1323131
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