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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Bibliografiset tiedot
Julkaisussa:Mol Ther Methods Clin Dev
Päätekijät: 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.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: American Society of Gene & Cell Therapy 2020
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Article
Linkit: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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