Lentiviral Transfer of γ-Globin with Fusion Gene NUP98-HOXA10HD Expands Hematopoietic Stem Cells and Ameliorates Murine β-Thalassemia

Recently, an engineered Homeobox-nucleoporin fusion gene, NUP98-HOXA10HD or NA10HD, was reported to expand and maintain murine hematopoietic stem cells (HSCs). We postulated that NA10HD would increase the number of human γ-globin-expressing cells to therapeutic levels. We developed a double gene len...

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Bibliografiske detaljer
Udgivet i:Mol Ther
Main Authors: Zhao, Hui Fen, Abraham, Allistair, Kim, Yoon-Sang, Wang, Yong-Dong, Pestina, Tamara, Zhan, Jun, Humphries, Keith, Nienhuis, Arthur W., Persons, Derek A.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2017
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Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5363206/
https://ncbi.nlm.nih.gov/pubmed/28190779
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.01.019
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