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

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 sil...

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Bibliographic Details
Main Authors: Zulema Romero, Beatriz Campo-Fernandez, Jennifer Wherley, Michael L Kaufman, Fabrizia Urbinati, Aaron R Cooper, Megan D Hoban, Kismet M Baldwin, Dianne Lumaquin, Xiaoyan Wang, Shantha Senadheera, Roger P Hollis, Donald B Kohn
Format: Article
Language:English
Published: Elsevier 2015-01-01
Series:Molecular Therapy: Methods & Clinical Development
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050116300249

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