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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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Udgivet i:Proc Natl Acad Sci U S A
Principais autores: Walsh, C E, Liu, J M, Xiao, X, Young, N S, Nienhuis, A W, Samulski, R J
Format: Artigo
Sprog:Inglês
Udgivet: National Academy of Sciences 1992
Fag:
Research Article
Online adgang:https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC49685/
https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/1323131/
https://ncbi.nlm.nih.govhttps://doi.org/10.1073/pnas.89.15.7257
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