Adeno-associated virus 2-mediated transduction and erythroid lineage-restricted long-term expression of the human beta-globin gene in hematopoietic cells from homozygous beta-thalassemic mice.

@article{Tan2001AdenoassociatedV2,
  title={Adeno-associated virus 2-mediated transduction and erythroid lineage-restricted long-term expression of the human beta-globin gene in hematopoietic cells from homozygous beta-thalassemic mice.},
  author={Man-Wah Tan and Keyun Qing and Shangzhen Zhou and Mervin C. Yoder and Arun Srivastava},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2001},
  volume={3 6},
  pages={940-6}
}
Adeno-associated virus 2 (AAV), a nonpathogenic human parvovirus, has gained attention as a potentially useful vector for human gene therapy. Here, we report successful AAV-mediated stable transduction and high-efficiency, long-term, erythroid lineage-restricted expression of a human beta-globin gene in primary murine hematopoietic stem cells in vivo. Bone marrow-derived primitive Sca-1(+), lin(-) hematopoietic stem cells from homozygous beta-thalassemic mice were transduced ex vivo with a… CONTINUE READING
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