Correction of a mouse model of sickle cell disease: lentiviral/antisickling beta-globin gene transduction of unmobilized, purified hematopoietic stem cells.

@article{Levasseur2003CorrectionOA,
  title={Correction of a mouse model of sickle cell disease: lentiviral/antisickling beta-globin gene transduction of unmobilized, purified hematopoietic stem cells.},
  author={Dana N. Levasseur and Thomas M Ryan and Kevin M. Pawlik and Tim M. Townes},
  journal={Blood},
  year={2003},
  volume={102 13},
  pages={
          4312-9
        }
}
Although sickle cell anemia was the first hereditary disease to be understood at the molecular level, there is still no adequate long-term treatment. Allogeneic bone marrow transplantation is the only available cure, but this procedure is limited to a minority of patients with an available, histocompatible donor. Autologous transplantation of bone marrow stem cells that are transduced with a stably expressed, antisickling globin gene would benefit a majority of patients with sickle cell disease… CONTINUE READING

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