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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...
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. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2020
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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 |
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