Creating New β-Globin-Expressing Lentiviral Vectors by High-Resolution Mapping of Locus Control Region Enhancer Sequences

Creating New β-Globin-Expressing Lentiviral Vectors by High-Resolution Mapping of Locus Control Region Enhancer Sequences

Hematopoietic stem cell gene therapy is a promising approach for treating disorders of the hematopoietic system. Identifying combinations of cis-regulatory elements that do not impede packaging or transduction efficiency when included in lentiviral vectors has proven challenging. In this study, we d...

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Bibliographic Details
Main Authors: Richard A. Morgan, Feiyang Ma, Mildred J. Unti, Devin Brown, Paul George Ayoub, Curtis Tam, Lindsay Lathrop, Bamidele Aleshe, Ryo Kurita, Yukio Nakamura, Shantha Senadheera, Ryan L. Wong, Roger P. Hollis, Matteo Pellegrini, Donald B. Kohn
Format: Article
Language:English
Published: Elsevier 2020-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
hemoglobin
SCD
sickle cell disease
MPRA
Massively Parrallel Reporter Assay
Shuffling
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120300668

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http://www.sciencedirect.com/science/article/pii/S2329050120300668

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