The human ankyrin 1 promoter insulator sustains gene expression in a β-globin lentiviral vector in hematopoietic stem cells

Lentiviral vectors designed for the treatment of the hemoglobinopathies require the inclusion of regulatory and strong enhancer elements to achieve sufficient expression of the β-globin transgene. Despite the inclusion of these elements, the efficacy of these vectors may be limited by transgene sile...

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Veröffentlicht in:Mol Ther Methods Clin Dev
Hauptverfasser: Romero, Zulema, Campo-Fernandez, Beatriz, Wherley, Jennifer, Kaufman, Michael L, Urbinati, Fabrizia, Cooper, Aaron R, Hoban, Megan D, Baldwin, Kismet M, Lumaquin, Dianne, Wang, Xiaoyan, Senadheera, Shantha, Hollis, Roger P, Kohn, Donald B
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Publishing Group 2015
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Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4445009/
https://ncbi.nlm.nih.gov/pubmed/26029723
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2015.12
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