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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Bibliografiska uppgifter
I publikationen:Mol Ther Methods Clin Dev
Huvudupphovsmän: 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.
Materialtyp: Artigo
Språk:Inglês
Publicerad: American Society of Gene & Cell Therapy 2020
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Article
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC7225380/
https://ncbi.nlm.nih.gov/pubmed/32426415
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.04.006
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