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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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Detalhes bibliográficos
Publicado no:Nucleic Acids Res
Principais autores: Ellis, J, Pasceri, P, Tan-Un, K C, Wu, X, Harper, A, Fraser, P, Grosveld, F
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 1997
Assuntos:
Acesso em linha: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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