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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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Detalles Bibliográficos
Autores principales: Morgan, Richard A., Ma, Feiyang, Unti, Mildred J., Brown, Devin, Ayoub, Paul George, Tam, Curtis, Lathrop, Lindsay, Aleshe, Bamidele, Kurita, Ryo, Nakamura, Yukio, Senadheera, Shantha, Wong, Ryan L., Hollis, Roger P., Pellegrini, Matteo, Kohn, Donald B.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7225380/
https://www.ncbi.nlm.nih.gov/pubmed/32426415
http://dx.doi.org/10.1016/j.omtm.2020.04.006