Evaluation of beta-globin gene therapy constructs in single copy transgenic mice.

Effective gene therapy constructs based on retrovirus or adeno-associated virus vectors will require regulatory elements that direct expression of genes transduced at single copy. Most beta-globin constructs designed for therapy of beta-thalassemias are regulated by the 5'HS2 component of the locus...

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Vydáno v:	Nucleic Acids Res
Hlavní autoři:	Ellis, J, Pasceri, P, Tan-Un, K C, Wu, X, Harper, A, Fraser, P, Grosveld, F
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	Oxford University Press 1997
Témata:	Research Article
On-line přístup:	https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC146564/ https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/9092642/ https://ncbi.nlm.nih.govhttps://doi.org/10.1093/nar/25.6.1296
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