Correction of murine sickle cell disease using gamma-globin lentiviral vectors to mediate high-level expression of fetal hemoglobin.

@article{Pestina2009CorrectionOM,
  title={Correction of murine sickle cell disease using gamma-globin lentiviral vectors to mediate high-level expression of fetal hemoglobin.},
  author={Tamara I. Pestina and Phillip W. Hargrove and Dennis W Jay and John Mark Nicholas Timm Gray and Kelli M Boyd and Derek A. Persons},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2009},
  volume={17 2},
  pages={245-52}
}
Increased levels of red cell fetal hemogloblin, whether due to hereditary persistence of expression or from induction with hydroxyurea therapy, effectively ameliorate sickle cell disease (SCD). Therefore, we developed erythroid-specific, gamma-globin lentiviral vectors for hematopoietic stem cell (HSC)-targeted gene therapy with the goal of permanently increasing fetal hemoglobin (HbF) production in sickle red cells. We evaluated two different gamma-globin lentiviral vectors for therapeutic… CONTINUE READING

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