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

@article{Zhao2017LentiviralTO,
  title={Lentiviral Transfer of γ-Globin with Fusion Gene NUP98-HOXA10HD Expands Hematopoietic Stem Cells and Ameliorates Murine β-Thalassemia.},
  author={Hui Fen Zhao and Allistair Abraham and Yoon-Sang Kim and Yong-Dong Wang and Tamara I. Pestina and Jun Zhan and Keith Humphries and A. W. Nienhuis and Derek A. Persons},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2017},
  volume={25 3},
  pages={593-605}
}
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 lentiviral vector encoding both human γ-globin and NA10HD… CONTINUE READING